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The Right Track—Living with Narcolepsy

Posted on by Charlie

Judy Orand knew something was wrong when she fell asleep at the wheel twice and spent her days sleeping. Her determination to find answers finally led to a narcolepsy diagnosis twenty years later.

Left to right: Judy Orand's husband, Pike Orand, and sons Ron and Randall Holahan.

Left to right: Judy Orand’s husband, Pike Orand, and sons Ron and Randall Holahan.

It’s hard to say when I first started having symptoms. I didn’t realize it was narcolepsy. It was when my grandmother was sick, and I was working in Plainview, Texas, in 1973 and 1974. That was the worst time, because I didn’t know I had it, but Mother found out and knew I was sick, and we went all over trying to find out what was wrong with me.

I was driving home after sitting up with Grandmother, and I guess I drove for 15 miles without knowing it. My headlights hitting the red triangle-warning sign behind a semi-truck parked on the side of the road woke me up. Otherwise I would have driven straight into that semi-truck. And then I had two or three more miles home.

A week or two later, I was driving a car full of kids and me home, and the windshield wipers put me to sleep, and we ended up in the ditch. And I forget who walked a mile to get the neighbor’s tractor to pull us out.

And then I kind of skipped a little while [with the narcolepsy] being so bad. I kind of lived a different life.

Left to right: Ron's partner, Lisa Heinemann, Ron, Kalyn, and Judy.

Left to right: Ron’s partner, Lisa Heinemann, Ron, Kalyn, and Judy.

Then, in 1974, ’75 and ’76, I slept. I had a recliner I slept in in the daytime and a bed at night, and the only other time I moved was to eat. My husband got upset about that. We lived by Scott and White Memorial Hospital, and they took us to see a famous neurologist and to a really good psychiatrist.

Both of them said I had narcolepsy, but the neurologist’s test proved it. And they both gave me medication that I was on for twenty years. I was on samples while they were doing the tests, and then I was on pills after the medication came on the market. So I was on it before it was a pill. With that pill, I don’t have a problem. It gives me energy. But without it, I’m useless.

I was probably a lot younger when I first had symptoms and just didn’t realize it. I wasn’t diagnosed at all until 1994. It really didn’t get bad until 1995. During that period, [my neurologist] was studying me to try and figure out what was going on, with stories from me, and my dreams.

I don’t have cataplexy. I do have sleep apnea, and I can’t use a CPAP machine. I think that makes my narcolepsy worse. I have a lot of memory gaps. I have fallen asleep standing up, but luckily, I woke up before I fell.

I always tell everybody that I grew up on the Santa Fe Railway. I spent my first seven years on my granddad’s farm. My mother and dad and I and my sisters lived in a little house my grandpa built for us. And life was good, until I was eight, and daddy took off. After that, Mother felt like she needed to get away. She was the first divorcee in town, and it was a little town.

Left to right: Judy, Ron, and Pike.

Left to right: Judy, Ron, and Pike.

So, we took the Santa Fe to Chicago and lived first with my aunt, and then, nearby, in our own apartment. And we stayed there seven years. Every summer, I got to go home and come back by myself on the Santa Fe. The first time I went on a Pullman; after that I took coach. So, I was bigger.

My grandparents got sick, and we came home. I was 16, and we came home to Lockney, Texas, out in the middle of the boondocks, in the panhandle, where my granddad had 360 irrigated acres. We thought he was poor ’til he died, and he was a rich man. Those 360 irrigated acres were like gold.

I have two beautiful boys. Randall was born in 1962, and my baby, Ron, was born in 1969. Randall lives right outside of Austin, and he’s been up a few times. Ron lives in Sioux City, Iowa, and he’s been down once.

I have a cat. Her name is Ginger, and we don’t know exactly how old she is. My daughter-in-law found her as a stray. When she feels like it, she uses the commode, and when she doesn’t feel like it, she uses her box or outside. I was just thinking this morning that we’ve had that cat for 17 years. But the way she was running around, you would have thought she was two years old—a baby—not an old lady.

I love my computer. I like books, but my eyes are so bad. I like TV. I like to talk. I used to garden, but I don’t do that anymore. I don’t have any time for things like that.

My husband is the main purpose of my life. We met in 1993 and married in 1994. He has been supposed to be dead at least five or six times, but he keeps on living. He had a bypass. He had five stents in one day in his heart. He had his aorta replaced and another stent, and then he had his aorta stented. Then, he got a pacemaker, a defibrillator, and prostate cancer. I’ve been there since almost all of it.

We’ve had tough times, but together, we have lots of fun. It doesn’t really matter if it’s watching TV. We have to go to the doctor a lot, and he hates it, so we make a game of it. We like to eat out, because neither one of us wants to do the dishes.

I love my family very much. Their support has kept me going all these years, along with great agencies like Caring Voice Coalition.

It took me twenty years to get my bachelor’s degree, because I was working. I had young children, and my mother was sick. And I had to keep dropping out and starting over. It’s supposed to take two years to get a master’s. It took me three years, because I dropped out a couple of times. But with my master’s, I almost got straight A’s, with a 3.88 GPA. I got an MBA, and I’m quite proud of it. It was in 2010, and I was well into my sixties.

My bachelor’s was in health care administration. I ran nursing homes. I’m a licensed Texas nursing home administrator, and I was a crisis manager. I would go into a very poorly run nursing home and turn it around in a year or so, and sit back and enjoy that for a year, and then move on to the next one. I did that for 13 years. A few times, I just worked for a few months to just help somebody out.

I enjoyed every minute of that work. I got attached, but sort of detached at the same time. You have to maintain a certain distance, but at the same, enough caring.

To those who have been recently diagnosed with narcolepsy, I would say, ‘Narcolepsy is something you have to live with. It’s something you have to be aware of. It’s kind of like being a diabetic. You have to follow the doctor’s orders. You have to eat right. You have to exercise right. And take your medicine right, and you will be normal for the 24-hour period. That’s long enough to get to the next one.

‘It’s not the end of the world. It’s not as bad as a lot of things. It has no pain, unless you do like me, and fall asleep standing up, and fall, or fall asleep driving, and wreck a car. So it’s very, very important that you follow the doctor’s instructions.’

I’m pretty persistent. I knew there was something wrong, so I kept after it and kept after it, and kept asking and kept asking—until I found the right person who told me what I needed to know.

Posted in Diseases, Featured, Media Center | 1 Comment

IYW: Sherry Gochenour on Living with Alpha-1

Posted on by Charlie

On the heels of her alpha-1 antitrypsin deficiency diagnosis, Sherry Gochenour found new direction in sharing what’s she’s learned about living with the disease.

I was working at a resort around February 2011, and I kept getting bronchitis, and I got pneumonia. I was going to my family doctor. He was giving me antibiotics and cough medicine and all the necessary medications to take care of it.

Sherry Gochenour and her sons (left to right) Christian, Dustin, and Josh.

Sherry Gochenour and her sons (left to right) Christian, Dustin, and Josh.

I’d get better, and a week later, it would come back. And this went on from February to probably about the middle of April.

My family doctor said, ‘Sherry, there’s something else going on here. We need to find out why you keep getting this.’

They sent me to a lung specialist at Rockingham Memorial Hospital in Harrisonburg, Virginia, and they did the blood work and X-rays. My lungs were two and a half sizes bigger than a normal person’s, and they did the Alpha-1 test, and it came back that I was a ZZ. (See sidebar, below)

That was May 2011, when I found out. I was in shock because I didn’t know what it was. I’d never heard of it before.

When the doctor told me I had lung disease, COPD, and emphysema, I was like, ‘You’ve got to be kidding me.’ I was an athlete. I ran track in high school. I have a school record in the two mile that can never be broken, because our high school combined with another high school my last year. I played softball from the age of 12 until I was 40. I loved volleyball, and I was a big swimmer.

I was just stopped dead in my tracks. I could hardly even walk up the stairs. It was awful.

There are a lot of speed bumps you have to go through with this. This disease is not known. There are no support groups around here for me.

My dad was a painter, and my ex-husband was in drywall, so I was around paint and drywall dust all of the time My lungs were pretty much damaged from all the chemicals and from having pneumonia as a child.

I worked at a printer; we made books. I was around a lot of paper dust that was flying around. Then, when I was diagnosed, I was cleaning condos, and you have some hardheaded people who want to mix chemicals. They were mixing bleach with other chemicals, and I would have to leave because the ammonia would get in my lungs and burn.

That would hurt anybody, but it affected me twice as badly. But I wasn’t diagnosed at the time, so I wasn’t aware of the damage it was doing.

The doctor put me on medical leave for about a month, right after I was diagnosed. When I started seeing the Alpha-1 doctor, they told me it was best that I did not work there, that it was not a good environment.

With my disability, I don’t know what I’d do, if it weren’t for CVC and Senator Mark Warner. I went to my doctor in October 2012, and said, ‘You know the severity of my lung condition. Do you think I’m a candidate for disability?’ He said, ‘Oh, yes, by all means. If you need anything, I’ll go write it out.’

I was denied twice for disability. I sent Senator Warner an email, and within three days he responded. He wrote me maybe ten letters. His office stayed in constant touch with me until I was granted. I didn’t even have to go before the judge.

I don’t think Alpha-1 patients should be denied disability. I’ve wanted to write our congressman about that. If an Alpha-1 doctor says an Alpha-1 patient should get it, then we should get it. There should be no questions asked.

[What I can do now] is very limited. I can go outside in the evenings after the humidity has gone and maybe water flowers and walk around in the yard. But it’s very limited. I went up to visit my friends, and we were sitting out on the deck, and I couldn’t breathe from being outside in the heat. I had to leave.

Some people look at it as a death sentence. I don’t look at it like that. I take one day at a time, because I could go out here today and get hit by a truck. Just look forward. Don’t look back.

I think the more knowledge you have about it, the better you understand. Since I found CVC and the Alpha-1 Association, I have found that there are tons of people out there like me.

The [lack of] knowledge is what gets me. People say, ‘Well, what’s wrong with you?’ And I’ll tell them. [And they’ll ask] ‘Well, what’s that?’ Even doctors and nurses at the hospital do that, and they’ll write down what I have, so they can go look it up.

“When the doctor told me I had lung disease, COPD, and emphysema, I was like, ‘You’ve got to be kidding me.’”

I think that I need to give back, and I try to think of ways of doing that and helping other people. CVC opened so many doors for me. I have made a lot of friends through CVC.

I’ve started my own Alpha-1 Facebook page, and I have over a hundred members now. There’s one man in particular who was diagnosed, and he’s not getting any help. I contacted him the other day, and I told him that there are organizations out there that will help him.

I told him about CVC. He was so happy that I contacted him about this. It feels like maybe this is my calling, to reach out to other Alphas to let them know that they’re not alone.

The biggest thing for me is the love for my kids. My three boys are the most precious things to me. They’re the main reason I fight this disease so hard.

When I was diagnosed, the doctor told there was a strong chance that my children were carriers. I have a 30-year-old son and a 25-year-old son. They’re very into eating right and working out. They’re both very healthy young men.

My 16-year-old was diagnosed when he was 12 or 13, and he is a carrier. He’s an MZ, and he was diagnosed with asthma when he was five. (See sidebar, below)

I have gone through so much with this as far as stress. I worry, if something does happen to me, who’s going to take care of my child? I know he’s 16 now, but I’ve even said to my other sons, ‘If something happens to me, you’ve got to promise me that you’re going to take care of him.’ They’ve guaranteed to me that they will see to it that he’s taken care of.

I even was at the point with mine that I dreaded my nurse coming here to do my infusions, because my veins were so scarred and battered that she could not find a vein. And I would break down and cry. I dreaded my infusion every week.

So I went to my doctor, and I said, ‘I just can’t take it anymore. I just can’t. It’s killing me.’

He asked, ‘Why don’t you have a port?’ And I said, ‘Let’s do it.’ I had a port put in May of last year, and, believe me, I don’t mind my treatments at all anymore.

It’s not pretty to look at, but you know what? I don’t care. I don’t have any pain. If I don’t have any pain, it doesn’t mean anything to me.

I don’t care what people think. When they ask, ‘Ew, why is that thing sticking out of your chest?’—I don’t care. Look all you want. If it’s bothering you all that badly, ask me about it. You feel like you have to explain what your disease is, because nobody’s heard of it.

Not only the public, but also medical staff needs to be educated on this disease. How are they supposed to help a patient if they don’t know what they’re dealing with?

Both my granddaddies supposedly died of tuberculosis. My dad’s father died when my dad was seven. My mom’s dad died when she was three weeks or three months old. My doctor now is questioning whether they died of tuberculosis. Back in the 1940s, they didn’t even know what Alpha-1 was.

Our youth concern me the most. If your child is diagnosed at a young age with asthma, it could be the onset of Alpha-1. Get them tested. It can help make life longer and [them] stronger if treated at a young age.

That’s why I had my son tested when I was diagnosed. He saw our Alpha-1 doctor last month—and his lungs are doing wonderfully. I went to see our doctor last month too, and he put me on a new inhaler, and I feel so much better than I did before.

If anybody wants to talk, drop me a line, I’ll talk to them. And if I could say anything to other patients, it would be, ‘Never give up hope. Never give up hope. And know that you’re never alone.’

Alpha-1 Antitrypsin Deficiency Genetics

Mutations in the SERPINA1 gene cause alpha-1 antitrypsin deficiency. The most common version (allele) of the SERPINA1 gene, called M, produces normal levels of alpha-1 antitrypsin. Most people in the general population have two copies of the M allele (MM) in each cell.

The S allele of the SERPINA1 gene produces moderately low levels of this protein, and the Z allele produces very little alpha-1 antitrypsin. Individuals with two copies of the Z allele (ZZ) in each cell are likely to have alpha-1 antitrypsin deficiency. Those with the SZ combination have an increased risk of developing lung diseases (such as emphysema), particularly if they smoke.

Individuals with an MS (or SS) combination usually produce enough alpha-1 antitrypsin to protect the lungs. People with MZ alleles, however, have a slightly increased risk of impaired lung or liver function.

From Genetics Home Reference, a service of the U.S. National Library of Medicine, National Institutes of Health

Posted in Diseases, Featured, Media Center | Leave a comment

Two New Treatments for IPF

Posted on by CVCinfo

FDA approves Esbriet to treat idiopathic pulmonary fibrosis.

October 15, 2014

The U.S. Food and Drug Administration today approved Esbriet (pirfenidone) for the treatment of idiopathic pulmonary fibrosis (IPF).

Idiopathic pulmonary fibrosis is a condition in which the lungs become progressively scarred over time. As a result, patients with IPF experience shortness of breath, cough, and have difficulty participating in everyday physical activities. Current treatments for IPF include oxygen therapy, pulmonary rehabilitation, and lung transplant.

“Esbriet provides a new treatment option for patients with idiopathic pulmonary fibrosis, a serious, chronic lung disease,” said Curtis J. Rosebraugh, M.D., M.P.H., director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research. “We continue to help advance medication therapies by approving products that treat conditions that impact public health.”

The FDA granted Esbriet fast track, priority review, orphan product, and breakthrough designations. Esbriet is being approved ahead of the product’s prescription drug user fee goal date of Nov. 23, 2014, the date the agency was scheduled to complete the review of the drug application.

Esbriet acts on multiple pathways that may be involved in the scarring of lung tissue. Its safety and effectiveness were established in three clinical trials of 1,247 patients with IPF. The decline in forced vital capacity – the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible – was significantly reduced in patients receiving Esbriet compared to patients receiving placebo.

Esbriet is not recommended for patients who have severe liver problems, end-stage kidney disease, or who require dialysis. Esbriet should be taken with food to minimize the potential for nausea and dizziness. Patients should avoid or minimize exposure to sunlight and sunlamps and wear sunscreen and protective clothing, as Esbriet may cause patients to sunburn more easily.

The most common side effects of Esbriet are nausea, rash, abdominal pain, upper respiratory tract infection, diarrhea, fatigue, headache, dyspepsia, dizziness, vomiting, decreased/loss of appetite, gastro-esophageal reflux disease, sinusitis, insomnia, decreased weight, and arthralgia.

The FDA also today approved Ofev (nintedanib) for the treatment of IPF.

Esbriet is manufactured for InterMune, Inc., Brisbane, California.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.




FDA approves Ofev to treat idiopathic pulmonary fibrosis.

October 15, 2014

The U.S. Food and Drug Administration today approved Ofev (nintedanib) for the treatment of idiopathic pulmonary fibrosis (IPF).

Idiopathic pulmonary fibrosis is a condition in which the lungs become progressively scarred over time. As a result, patients with IPF experience shortness of breath, cough, and have difficulty participating in everyday physical activities. Current treatments for IPF include oxygen therapy, pulmonary rehabilitation, and lung transplant.

“Today’s Ofev approval expands the available treatment options for patients with idiopathic pulmonary fibrosis, a serious, chronic condition,” said Mary H. Parks, M.D., deputy director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research. “Providing health care professionals and patients with additional treatment options helps enable appropriate care decisions based on a patient’s need.”

The FDA granted Ofev fast track, priority review, orphan product, and breakthrough designations. Ofev is being approved ahead of the product’s prescription drug user fee goal date of Jan. 2, 2015, the date the agency was scheduled to complete the review of the drug application.

Ofev is a kinase inhibitor that blocks multiple pathways that may be involved in the scarring of lung tissue. Its safety and effectiveness were established in three clinical trials of 1,231 patients with IPF. The decline in forced vital capacity – the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible – was significantly reduced in patients receiving Ofev compared to patients receiving placebo.

Ofev is not recommended for patients who have moderate to severe liver problems. Ofev can cause birth defects or death to an unborn baby. Women should not become pregnant while taking Ofev. Women who are able to get pregnant should use adequate contraception during and for at least three months after the last dose of Ofev.

The most common side effects of Ofev are diarrhea, nausea, abdominal pain, vomiting, liver enzyme elevation, decreased appetite, headache, decreased weight, and high blood pressure.

The FDA also today approved Esbriet (pirfenidone) for the treatment of IPF.

Ofev is distributed by Boehringer Ingelheim Pharmaceuticals, Inc., Ridgefield, Connecticut.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.




InterMune Announces Expanded Access Program for Pirfenidone to Treat Idiopathic Pulmonary Fibrosis (IPF) in the United States

Date(s): 5/16/14 8:00 AM

For a complete listing of InterMune news releases, please click here

BRISBANE, Calif., May 16, 2014 /PRNewswire/ — InterMune, Inc. (NASDAQ: ITMN) today announced it will provide compassionate use of pirfenidone through a multi-center Expanded Access Program (EAP) in the United States to be conducted under InterMune’s U.S. IND. Pirfenidone is an investigational therapy in the U.S. and has not been approved by the U.S. Food and Drug Administration (FDA).

Expanded access programs provide a mechanism for early access to an investigational drug in the pre-approval period to treat patients with a serious or immediately life-threatening disease or condition that has no comparable or satisfactory alternative treatment options.

“We are pleased to offer this expanded access program for eligible patients in the U.S.,” said Jonathan Leff, M.D., Executive Vice President of Research and Development, InterMune. “This EAP provides a mechanism for eligible patients to access pirfenidone as a treatment option, following the recent successful completion of our ASCEND Phase 3 trial and prior to FDA’s final decision on the approvability of pirfenidone in the United States.”

To enroll in the EAP, a patient must meet specific clinical criteria. Eligible patients must have a clinical and radiographic diagnosis of IPF with the presence of a usual interstitial pneumonia (UIP) pattern on high-resolution computed tomography (HRCT). Additional criteria for the EAP are listed on It is important to note that only a physician who is participating in the EAP can assess a potential patient for eligibility. The EAP protocol contains provisions for stopping enrollment of patients in the EAP upon a decision by the FDA on the approvability of a pirfenidone New Drug Application (NDA).

There are currently a limited number of sites accepting patients for enrollment to the pirfenidone EAP and InterMune expects that all sites will be participating by September of 2014. InterMune is working with the Pulmonary Fibrosis Foundation (PFF), the Coalition for Pulmonary Fibrosis (CPF) and other advocacy groups to enable patients with IPF to obtain information about the pirfenidone EAP.

For more information about the pirfenidone EAP, including eligibility criteria and participating clinical centers, contact InterMune Medical Information at 888-486-6411 or the Pulmonary Fibrosis Foundation (PFF) at 844-TalkPFF (844-825-5733) or visit

About IPF
Idiopathic pulmonary fibrosis (IPF) is an irreversible and ultimately fatal disease characterized by progressive loss of lung function due to fibrosis (scarring) in the lungs, which hinders the ability of lungs to absorb oxygen. IPF inevitably causes shortness of breath, and a deterioration in lung function and exercise tolerance. IPF patients follow different and unpredictable clinical courses and it is not possible to predict if a patient will progress slowly or rapidly, or when the rate of decline may change. Periods of transient clinical stability in IPF, when they occur, inevitably give way to continued disease progression. The median survival time from diagnosis is two to five years, with a five-year survival rate of approximately 20-40 percent, which makes IPF more rapidly lethal than many malignancies, including breast, ovarian and colorectal cancers. IPF typically occurs in patients over age 45, and tends to affect slightly more men than women.

About Pirfenidone
Pirfenidone is an orally active, anti-fibrotic agent that inhibits the synthesis of TGF-beta, a chemical mediator that controls many cell functions including proliferation and differentiation, and plays a key role in fibrosis. Pirfenidone also inhibits the synthesis of TNF-alpha, a cytokine that is known to have an active role in inflammation.

About InterMune
InterMune is a biotechnology company focused on the research, development and commercialization of innovative therapies in pulmonology and orphan fibrotic diseases. In pulmonology, the company is focused on therapies for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive, irreversible, unpredictable and ultimately fatal lung disease. Pirfenidone is not approved for marketing in the United States. InterMune’s research programs are focused on the discovery of targeted, small-molecule therapeutics and biomarkers to treat and monitor serious pulmonary and fibrotic diseases. For additional information about InterMune and its R&D pipeline, please visit

Forward-Looking Statements
This news release contains forward-looking statements within the meaning of section 21E of the Securities Exchange Act of 1934, as amended, that reflect InterMune’s judgment and involve risks and uncertainties as of the date of this release, including without limitation InterMune’s expectations regarding the availability of its Expanded Access Program for patients in the U.S. with IPF. All forward-looking statements and other information included in this press release are based on information available to InterMune as of the date hereof, and InterMune assumes no obligation to update any such forward-looking statements or information. InterMune’s actual results could differ materially from those described in InterMune’s forward-looking statements.

Other factors that could cause or contribute to such differences include, but are not limited to, those discussed in detail under the heading “Risk Factors” in InterMune’s most recent annual report on Form 10-K filed with the Securities and Exchange Commission (SEC) on February 24, 2014 (the “Form 10-K”) and other periodic reports filed with the SEC, including but not limited to the following: (i) the risks related to the uncertain, lengthy and expensive clinical development process for the company’s product candidates, including having no unexpected safety, toxicology, clinical or other issues and having no unexpected clinical trial results such as unexpected new clinical data and unexpected additional analysis of existing clinical data; (ii) risks related to the regulatory process for the company’s product candidates, including the possibility that the results of the new 52-week Phase 3 clinical trial (ASCEND) having an FVC endpoint may not be satisfactory to the FDA for InterMune to receive regulatory approval for pirfenidone in the United States; (iii) risks related to unexpected regulatory actions or delays or government regulation generally; and (iv) risks related to the company’s manufacturing strategy, which relies on third-party manufacturers and which exposes InterMune to additional risks where it may lose potential revenue. The risks and other factors discussed above should be considered only in connection with the fully discussed risks and other factors discussed in detail in the Form 10-K and InterMune’s other periodic reports filed with the SEC, all of which are available via InterMune’s web site at

SOURCE InterMune, Inc.

Investors: Jim Goff, InterMune, Inc., 415-466-2228,, Media: Geoff Curtis, Edelman, 312-550-8138,



Lundbeck’s SABRIL® (vigabatrin) Now Approved by U.S. FDA as an Adjunctive Treatment Option for Children 10 and older with Refractory Complex Partial Seizures

Deerfield, Ill., October 28, 2013 – The U.S. Food and Drug Administration (FDA) approved SABRIL (vigabatrin) as add-on therapy for the treatment of refractory complex partial seizures (CPS) in children 10 years of age and older who have inadequately responded to several other treatments and if the possible benefit outweighs the risk of vision loss.1 This approval expands upon the age range of SABRIL’s previous indication as adjunctive therapy for adults with refractory CPS. SABRIL is not indicated as a first-line agent for refractory CPS.

Of the more than two million Americans affected by epilepsy,2 approximately 35 percent have CPS, which originates from a single region of the brain and can cause impaired consciousness.3 Approximately 30 to 36 percent of those with CPS continue to have seizures despite trying multiple therapies, and are considered to have refractory CPS.4,5,6

“It is crucially important for people with challenging seizures like refractory CPS to not give up and continue striving for improved seizure management, and this expanded Sabril indication provides another consideration for the treatment of those ten and older with refractory CPS,” said Philip Gattone, president and CEO of the Epilepsy Foundation. “We encourage people living with such challenging seizures and their loved ones to have ongoing conversations with their doctor about available options to help manage this intractable seizure disorder.”

When SABRIL was first approved in 2009, a patient registry was established to collect information on all patients who are prescribed SABRIL. To date, more than 5,600 patients have been treated with SABRIL, a substantial number of whom have been treated for refractory CPS.7 In evaluating whether to start SABRIL, doctors, patients and their caregivers work together to assess the risk of permanent vision loss versus the benefit of seizure reduction. There are other serious risks associated with SABRIL. Please see the important safety information below for more details.

“With so many children still having seizures due to refractory CPS, we are very pleased that the FDA has approved SABRIL for patients 10 and older who may benefit from a new add-on treatment option,” said Amy Magro, Director of Epilepsy Marketing at Lundbeck. “For those caring for a child as young as 10, we hope this new indication provides encouragement to speak with their child’s doctor about the risks and potential benefits of adding SABRIL for refractory CPS.”

In addition to its refractory CPS indication, SABRIL is approved for use in babies one month to two years of age with infantile spasms if the possible benefit outweighs the potential risk of vision loss.

For more information, please visit

About SABRIL® (vigabatrin) 1
SABRIL is a prescription oral antiepileptic drug developed in the United States by Lundbeck. SABRIL is available in 500-mg tablets or 500-mg packets of powder for oral suspension. Because of the risk of permanent vision loss, SABRIL is available only through a restricted program under a REMS called the SHARE Program. (1-888-45-SHARE).

SABRIL (vigabatrin) is a prescription medicine used with other treatments in adults and children 10 years of age and older with refractory complex partial seizures (CPS), who have not responded well enough to several other treatments, and if the possible benefits outweigh the risk of vision loss. SABRIL should not be the first medicine used to treat CPS.

SABRIL (vigabatrin) is a prescription medicine used in babies, 1 month to 2 years old, with infantile spasms (IS), if the possible benefits outweigh the possible risk of vision loss.

Important Safety Information


See Medication Guide and full Prescribing Information for complete information

In all people who take SABRIL:

• You are at risk for vision loss with any amount of SABRIL

• Your risk of vision loss may be higher the more SABRIL you take daily and the longer you take it

• It is not possible for your healthcare provider to know when vision loss will happen. It could happen soon after starting SABRIL or any time during treatment. It may even happen after treatment has stopped.

Please see SABRIL Medication Guide, full Prescribing Information including Boxed Warning, and Instructions for Use; go to, or call toll-free 1-888-45-SHARE (1-888-457-4273).

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit, or call 1-800-FDA-1088.

About Lundbeck in the U.S.
A wholly owned subsidiary of H. Lundbeck A/S of Denmark, Lundbeck in the United States is headquartered in Deerfield, Illinois, and is committed to providing innovative specialty therapies that fulfill unmet medical needs of people with central nervous system (CNS) disorders, including several therapies for people with challenging seizure disorders.

With a special commitment to the epilepsy community, Lundbeck actively supports and participates in hundreds of community-based initiatives. Learn more about our epilepsy community programs at

About H. Lundbeck A/S
Lundbeck is a global pharmaceutical company highly committed to improving the quality of life of people living with brain diseases. For this purpose, Lundbeck is engaged in the entire value chain throughout research, development, production, marketing and sales of pharmaceuticals across the world. The company’s products are targeted at disorders such as depression and anxiety, psychotic disorders, epilepsy, Huntington’s, Alzheimer’s and Parkinson’s diseases. Lundbeck’s pipeline consists of several mid- to late-stage development programs.

Lundbeck employs more than 5,800 people worldwide, 2,000 of whom are based in Denmark. We have employees in 57 countries and our products are registered in more than 100 countries. We have research centers in Denmark, China and the United States and production facilities in Italy, France, Mexico, China and Denmark. Lundbeck generated revenue of approximately DKK 15 billion in 2012. Lundbeck’s shares are listed on the stock exchange in Copenhagen under the symbol “LUN.” Lundbeck has a sponsored Level 1 ADR programme listed in the US (OTC) under the symbol “HLUYY.” For additional information, we encourage you to visit our corporate site


  1. SABRIL® (vigabatrin) full Prescribing Information, Deerfield, IL. Lundbeck. 2013.
  2. Epilepsy Foundation. About Epilepsy: Statistics. Accessed 10/2/13.
  3. Carroll E. Medscape. Complex Partial Seizures. Accessed 10/2/13.
  4. Kwan P, Brodie MJ. Early identification of refractory epilepsy. N Engl J Med. 2000; 342:314-9.
  5. Devinsky O. Patients with refractory seizures. N Engl J Med. 1999;340:1565-70.
  6. Rielo DM. Medscape. Vagus Nerve Stimulation. 2011. Accessed 10/2/13.
  7. Data on File.


Matt Flesch

SABRIL is a registered trademark of Lundbeck.



Actelion receives U.S. FDA approval of Opsumit (macitentan) for the treatment of pulmonary arterial hypertension.

Allschwil, Switzerland, October 18, 2013 – Actelion Ltd (SIX: ATLN) announced today that the United States Food and Drug Administration (FDA) has approved the use of the orally available endothelin receptor antagonist Opsumit® (macitentan) 10 mg once daily for the treatment of pulmonary arterial hypertension (PAH) to delay disease progression.

Opsumit is indicated for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids, or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). Opsumit also reduced hospitalization for PAH.

Effectiveness was established in a long-term study in PAH patients with predominantly WHO Functional Class II-III symptoms treated for an average of 2 years. Patients were treated with Opsumit® monotherapy or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids. Patients had idiopathic and heritable PAH (57%), PAH caused by connective tissue disorders (31%), and PAH caused by congenital heart disease with repaired shunts (8%).

Dr. Vallerie McLaughlin, Director of the Pulmonary Hypertension Program in the Division of Cardiovascular Medicine at the University of Michigan, commented: “Over the past twenty years, great strides have been made in treating PAH patients. However, there has been a medical need for innovative treatments that improve long-term outcomes. Opsumit® is the first clinically proven and only oral treatment option indicated to delay disease progression and reduce the need for PAH hospitalization.”

Dr. McLaughlin concluded: “These effects were demonstrated in SERAPHIN, the first and largest PAH outcome study to date, where Opsumit® was given on average for 2 years, as a monotherapy or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids. I am very pleased that PAH patients will have this new treatment option.”

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: “Today’s approval of Opsumit® by the FDA is providing the PAH community with a unique treatment option, the only oral PAH medicine that has proven to delay disease progression. Over the last 14 years, Actelion has worked tirelessly to first discover and then develop Opsumit® in the largest, longest and first-ever outcome study in PAH. I would like to express my gratitude to all the members of the PAH community. Without their contribution, Opsumit® would not have become a reality. We will now leverage our existing PAH expertise and infrastructure to bring Opsumit® to patients within the coming weeks.”

The US label for Opsumit® carries a Boxed Warning alerting patients and health care professionals that the drug should not be used in pregnant women because it can harm the developing fetus. Female patients can receive the drug only through the Opsumit REMS Program. All female patients must be enrolled in the program, comply with pregnancy testing requirements and be counselled regarding the need for contraception.

The most common adverse reactions (more frequent than placebo by 3% or more) observed in patients treated with Opsumit® were anemia, nasopharyngitis/pharyngitis, bronchitis, headache, influenza, and urinary tract infection.

Physicians are advised to measure hemoglobin and liver enzymes prior to initiation of Opsumit® and repeat during treatment as clinically indicated.

In the United States, Actelion expects Opsumit® to become available to patients in November. Outside of the United States, Actelion continues to work with health authorities to obtain regulatory approval for Opsumit® .

The FDA approval was based in part on data from the landmark phase III SERAPHIN study. Published in the New England Journal of Medicine in August 2013, the SERAPHIN study showed the risk of the first occurrence of a morbidity or mortality event, the primary endpoint of the study, was reduced by 45% (p<0.0001) with macitentan 10 mg compared to placebo. This effect was observed irrespective of whether or not patients were already treated with other therapies for PAH. SERAPHIN also showed a risk reduction in PAH related hospitalization and death of 50% (p<0.0001) compared to placebo. [1].

Pulmonary arterial hypertension (PAH) is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual. The symptoms of PAH are non-specific and can range from mild breathlessness and fatigue during normal daily activity to symptoms of right heart failure and severe restrictions on exercise capacity and ultimately reduced life expectancy.


Opsumit® (macitentan) is a novel dual endothelin receptor antagonist (ERA) that resulted from a tailored drug discovery process with the target of developing an ERA to address efficacy and safety [3].

SERAPHIN (Study with an Endothelin Receptor Antagonist in Pulmonary arterial Hypertension to Improve cliNical outcome) was the largest and longest randomized, controlled study in PAH patients to include a clearly defined morbidity/mortality primary endpoint [2]. The pivotal Phase III study was designed to evaluate the efficacy and safety of Opsumit®(macitentan) – a novel dual endothelin receptor antagonist that resulted from a tailored drug discovery process – through the primary endpoint of time to first morbidity and all-cause mortality event in patients with symptomatic PAH.

Global enrollment was completed in December 2009 with a total of 742 patients. Patients were randomized 1:1:1 to receive two different doses of macitentan (3 mg and 10 mg once daily) or placebo. Patients were allowed to receive PAH background therapy throughout the study, either PDE-5 inhibitors or oral/inhaled prostanoids. This event-driven study was conducted in 151 centers from almost 40 countries in North America, Latin America, Europe, Asia-Pacific and Africa and was completed in the first half of 2012, with 287 patients having an adjudicated event.

Dr. McLaughlin is a consultant to Actelion and was an investigator in the SERAPHIN trial.

Patients were randomized to placebo (n=250), macitentan 3 mg (n=250), or macitentan 10 mg (n=242). The primary end point occurred in 46.4%, 38.0%, and 31.4% of the patients in these groups, respectively. The hazard ratio for macitentan 3 mg versus placebo was 0.70 (97.5% CI, 0.52 to 0.96; p=0.0108) and the hazard ratio for macitentan 10 mg versus placebo was 0.55 (97.5% CI, 0.39 to 0.76; p<0.0001). Worsening of pulmonary arterial hypertension was the most frequent primary end point event. The effect of macitentan on this end point was observed irrespective of background therapy for pulmonary arterial hypertension. [1]

Opsumit is contraindicated in pregnancy because it may harm the developing fetus. Females of reproductive potential should be counselled on the use of reliable contraception and have a negative pregnancy test prior to initiating therapy and monthly thereafter.

Other ERAs have been associated with elevations of aminotransferases, hepatotoxicity, and liver failure. Liver enzyme tests should be obtained prior to initiation of Opsumit® and repeated during treatment as clinically indicated. If clinically relevant aminotransferase elevations occur, or if elevations are accompanied by clinical symptoms of hepatoxicity, discontinue Opsumit®.

Decreases in hemoglobin concentration and hematocrit occurred following administration of other ERAs and were observed in clinical studies with OPSUMIT. The decreases occurred early and stabilized thereafter. Decreases in hemoglobin seldom require transfusion. Initiation of Opsumit® is not recommended in patients with severe anemia. Hemoglobin should be measured prior to initiation of treatment and repeat during treatment as clinically indicated.

Should signs of pulmonary edema occur, consider the possibility of associated PVOD. If confirmed, discontinue Opsumit®.

Other ERAs have been associated with adverse effects on spermatogenesis. Men should be counseled about potential effects on fertility.

The use of Opsumit® with strong CYP3A4 inducers or inhibitors should be avoided.

The most common adverse reactions (more frequent than placebo by 3% or more) observed in patients treated with Opsumit were anemia, nasopharyngitis/pharyngitis, bronchitis, headache, influenza, and urinary tract infection.


Approval of the new drug application for Opsumit® (macitentan) was issued by the US Food and Drug Administration (FDA) on 18October 2013 for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids, or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). The need for PAH hospitalization was also reduced.

Regulatory reviews are ongoing in Europe, Canada, Switzerland, Australia, Taiwan, Korea and Mexico.


Pulmonary arterial hypertension (PAH) is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual. The symptoms of PAH are non-specific and can range from mild breathlessness and fatigue during normal daily activity to symptoms of right heart failure and severe restrictions on exercise capacity and ultimately reduced life expectancy.

PAH is one group within the classification of pulmonary hypertension (PH). This group includes idiopathic PAH, heritable PAH and PAH caused by factors which include connective tissue disease, HIV infection and congenital heart disease.

The last decade has seen significant advances in the understanding of the pathophysiology of PAH, which has been paralleled with developments of treatment guidelines and new therapies. Drugs targeting the three pathways that have been established in the pathogenesis of PAH are endothelin receptor antagonists (ERAs), prostacyclins and phosphodiesterase-5 inhibitors. PAH treatments have transformed the prognosis for PAH patients from symptomatic improvements in exercise tolerance 10 years ago to delayed disease progression today. Improved disease awareness and evidence-based guidelines developed from randomized controlled clinical trial data have highlighted the need for early intervention, goal-oriented treatment and combination therapy.

In PAH, survival rates are unacceptably low and PAH remains incurable.


  1. Pulido T et al. Macitentan and Morbidity and Mortality in Pulmonary Arterial Hypertension. N Engl J Med 2013;369:809-18.
  2. Proceedings of the 4th world symposium on pulmonary hypertension. J Am CollCardiol 2009;54(1 Suppl).
  3. Bolli MH et al. The Discovery of N-[5-(4-Bromophenyl)-6-[2-[(5-bromo-2-pyrimidinyl)oxy]ethoxy]-4-pyrimidinyl]-N’-propylsulfamide (Macitentan), an Orally Active, Potent Dual Endothelin Receptor Antagonist. J Med Chem. 2012; 55:7849-61.
  4. Gatfield J, Mueller Grandjean C, Sasse T, Clozel M, Nayler O (2012). Slow Receptor Dissociation Kinetics Differentiate Macitentan from Other Endothelin Receptor Antagonists in Pulmonary Arterial Smooth Muscle Cells. PLOS ONE 7(10): e47662. doi:10.1371/journal.pone.0047662
  5. Iglarz M et al. Pharmacology of macitentan, an orally active tissue-targeting dual endothelin receptor antagonist. J PharmacolExpTher. 2008;327(3):736-45.
  6. Sidharta PN et al. Macitentan: Entry-into-humans study with a new endothelin receptor antagonist. Eur J ClinPharmacol. 2011;67(10):977-84
  7. Bruderer S et al. Absorption, distribution, metabolism, and excretion of macitentan, a dual endothelin receptor antagonist, in humans. Xenobiotica. 2012 Sep;42(9):901-10
  8. Bruderer S et al. Effect of cyclosporine A and rifampin on the pharmacokinetics of macitentan, a tissue-targeting dual endothelin receptor antagonist. AAPS J. 2012;14(1):68-78.
  9. Galiè N, Hoeper MM, Humbert M, et al; ESC Committee for Practice Guidelines (CPG). Guidelines for the diagnosis and treatment of pulmonary hypertension: the Task Force for the Diagnosis and Treatment of Pulmonary Hypertension of the European Society of Cardiology (ESC) and the European Respiratory Society (ERS), endorsed by the International Society of Heart and Lung Transplantation (ISHLT). Eur Heart J 2009;30:2493-537
  10. Benza RL, Miller DP, Barst RJ, Badesch DB, Frost AE, McGoon MD. An evaluation of long-term survival from time of diagnosis in pulmonary arterial hypertension from REVEAL. Chest 2012;142:448-56.

Actelion Ltd
Actelion Ltd is a biopharmaceutical company with its corporate headquarters in Allschwil/Basel, Switzerland. Actelion’s first drug Tracleer® (bosentan), an orally available dual endothelin receptor antagonist, has been approved as a therapy for pulmonary arterial hypertension. Actelion markets Tracleer through its own subsidiaries in key markets worldwide, including the United States (based in South San Francisco), the European Union, Japan, Canada, Australia and Switzerland. Actelion, founded in late 1997, is a leading player in innovative science related to the endothelium – the single layer of cells separating every blood vessel from the blood stream. Actelion’s over 2,300 employees focus on the discovery, development and marketing of innovative drugs for significant unmet medical needs. Actelion shares are traded on the SIX Swiss Exchange (ticker symbol: ATLN) as part of the Swiss blue-chip index SMI (Swiss Market Index SMI®).

For further information please contact:
Roland Haefeli
Senior Vice President, Head of Investor Relations & Public Affairs
Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil
+41 61 565 62 62
+1 650 624 69 36

The above information contains certain “forward-looking statements”, relating to the company’s business, which can be identified by the use of forward-looking terminology such as “estimates”, “believes”, “expects”, “may”, “are expected to”, “will”, “will continue”, “should”, “would be”, “seeks”, “pending” or “anticipates” or similar expressions, or by discussions of strategy, plans or intentions. Such statements include descriptions of the company’s investment and research and development programs and anticipated expenditures in connection therewith, descriptions of new products expected to be introduced by the company and anticipated customer demand for such products and products in the company’s existing portfolio. Such statements reflect the current views of the company with respect to future events and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements of the company to be materially different from any future results, performances or achievements that may be expressed or implied by such forward-looking statements. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected.


News Release Intended for U.S. Media Only

FDA Approves Bayer’s New Class of Drug Adempas® (riociguat) tablets to Treat Adults with PAH and Persistent, Recurrent or Inoperable CTEPH First and only drug approved in U.S. to Treat Two Forms of Pulmonary Hypertension (WHO Group 1 and 4)

Whippany, N.J., October 8, 2013 – Bayer HealthCare announced today that the United States Food and Drug Administration (FDA) has approved Adempas®  (riociguat) tablets for: (i) the treatment of adults with persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH) (WHO*  Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class; and (ii) the treatment of adults with pulmonary arterial hypertension (PAH) (WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening. In PAH, efficacy was shown in patients on Adempas monotherapy or in combination with endothelin receptor antagonists (ERAs) or prostanoids (inhaled, oral or subcutaneous). Studies establishing effectiveness included predominately patients with WHO functional class II-III and etiologies of idiopathic or heritable PAH (61%) or PAH associated with connective tissue diseases (25%). Adempas is the only treatment approved in the U.S. for use in two types of pulmonary hypertension (WHO Group 1 and 4). It is the first and only FDA-approved drug therapy for persistent/recurrent CTEPH after surgical treatment or inoperable CTEPH. It is also the only approved oral therapy in PAH with efficacy shown in monotherapy or in combination with ERAs or prostanoids. For all female patients, Adempas is available only through a restricted program called the Adempas Risk Evaluation and Mitigation Strategy (REMS) Program.

1 World Health Organization

“CTEPH and PAH are serious and life-threatening diseases,” said Nick H. Kim, Associate Clinical Professor of Medicine, Division of Pulmonary and Critical Care Medicine; Director, Pulmonary Vascular Medicine; Director, Fellowship Program; University of California San Diego. “The approval of Adempas equips physicians with a new approach to treating PAH patients, and it gives us the first approved drug treatment for patients with inoperable CTEPH or with persistent/recurrent CTEPH after surgery. While surgery should always be considered as the first treatment option for CTEPH, the fact remains that up to forty percent of CTEPH patients are not eligible for surgery, and ten to thirty-five percent of CTEPH patients have disease that persists after surgery.” PAH is a disease characterized by elevated pressure in the pulmonary arteries. CTEPH is a form of pulmonary hypertension in which blood clots and thromboembolic occlusion of the pulmonary vessels leads to increased pressure in the pulmonary arteries. The standard treatment for CTEPH is pulmonary endarterectomy, a potentially curative surgery that clears clots and scar material from the blood vessels of the lung. “Bayer is deeply committed to bringing new treatment options to patients with life-threatening diseases. Adempas is an excellent example of this commitment, because it is the result of years of dedicated research in our Bayer laboratories into a new way of treating two forms of pulmonary hypertension,” said Pamela A. Cyrus, MD, Vice President and Head, U.S. Medical, Bayer HealthCare Pharmaceuticals. “We are pleased to bring this new class of treatment to patients with PAH or with inoperable CTEPH or persistent/recurrent CTEPH after surgical treatment.” Rino Aldrighetti, President and CEO, Pulmonary Hypertension Association added, “From a patient’s perspective, living with pulmonary hypertension remains difficult. We know that not all treatments work for all people. We get excited when there is a new treatment option for PAH patients, and we are thrilled there is finally an approved drug treatment for people living with persistent/recurrent CTEPH after surgical treatment or inoperable CTEPH.” Adempas, a stimulator of soluable guanylate cyclase (sGC), represents a new class of drug now available in the U.S. Pulmonary hypertension is associated with endothelial dysfunction, impaired synthesis of nitric oxide (NO) and insufficient stimulation of the NO-sGC-cGMP pathway. Adempas sensitizes sGC to endogenous NO by stabilizing the NO-sGC binding. Adempas also directly stimulates sGC via a different binding site independently of NO. Adempas restores the NO-sGC-cGMP pathway and leads to increased generation of cGMP with subsequent vasodialation. The most common adverse reactions occurring more frequently (>3%) on Adempas than placebo were headache (27% vs 18%), dyspepsia/gastritis (21% vs. 8%), dizziness (20% vs 13%), nausea (14% vs 11%), diarrhea (12% vs 8%), hypotension (10% vs 4%), vomiting (10% vs 7%), anemia (7% vs 2%), gastroesophageal reflux disease (5% vs 2%), and constipation (5% vs 1%). Other events that were seen more frequently in Adempas compared to placebo and potentially related to treatment were: palpitations, nasal congestion, epistaxis, dysphagia, abdominal distension and peripheral edema. About Patient Assistance Program Bayer offers patient assistance through the Adempas Aim Support Center program, which will assist with obtaining coverage and patient support of Adempas. Patients and providers may contact the program at 1-855-4ADEMPAS for additional information. IMPORTANT SAFETY INFORMATION 

WARNING: EMBRYO-FETAL TOXICITY Do not administer Adempas (riociguat) tablets to a pregnant female because it may cause fetal harm.

Females of reproductive potential: Exclude pregnancy before the start of treatment, monthly during treatment, and 1 month after stopping treatment. Prevent pregnancy during treatment and for one month after stopping treatment by using acceptable methods of contraception.

For all female patients, Adempas is available only through a restricted program called the Adempas Risk Evaluation and Mitigation Strategy (REMS) Program.

Contraindications. Adempas is contraindicated in:

Warnings and Precautions Embryo-Fetal Toxicity. Adempas may cause fetal harm when administered during pregnancy and is contraindicated for use in women who are pregnant. In females of reproductive potential, exclude pregnancy prior to initiation of therapy, advise use of acceptable contraception and obtain monthly pregnancy tests. For females, Adempas is only available through a restricted program under the Adempas REMS Program. Adempas REMS Program. Females can only receive Adempas through the Adempas REMS Program, a restricted distribution program. Important requirements of the Adempas REMS program include the following:

Further information, including a list of certified pharmacies, is available at or 1-855-4ADEMPAS. Hypotension. Adempas reduces blood pressure. Consider the potential for symptomatic hypotension or ischemia in patients with hypovolemia, severe left ventricular outflow obstruction, resting hypotension, autonomic dysfunction, or concomitant treatment with antihypertensives or strong CYP and P-gp/BCRP inhibitors. Consider a dose reduction if patient develops signs or symptoms of hypotension. Bleeding. In the placebo-controlled clinical trials program, serious bleeding occurred in 2.4% of patients taking Adempas compared to 0% of placebo patients. Serious hemoptysis occurred in 5 (1%) patients taking Adempas compared to 0 placebo patients, including one event with fatal outcome. Serious hemorrhagic events also included 2 patients with vaginal hemorrhage, 2 with catheter site hemorrhage, and 1 each with subdural hematoma, hematemesis, and intra-abdominal hemorrhage. Pulmonary Veno-Occlusive Disease. Pulmonary vasodilators may significantly worsen the cardiovascular status of patients with pulmonary veno-occlusive disease (PVOD). Therefore, administration of Adempas to such patients is not recommended. Should signs of pulmonary edema occur, the possibility of associated PVOD should be considered and if confirmed, discontinue treatment with Adempas.  Most Common Adverse Reactions  The most common adverse reactions occurring more frequently (>3%) on Adempas than placebo were headache (27% vs 18%), dyspepsia/gastritis (21% vs. 8%), dizziness (20% vs 13%), nausea (14% vs 11%), diarrhea (12% vs 8%), hypotension (10% vs 4%), vomiting (10% vs 7%), anemia (7% vs 2%), gastroesophageal reflux disease (5% vs 2%), and constipation (5% vs 1%). Other events that were seen more frequently in Adempas compared to placebo and potentially related to treatment were: palpitations, nasal congestion, epistaxis, dysphagia, abdominal distension and peripheral edema. For important risk and use information, please see the full Prescribing Information, including Boxed Warning, at About Bayer HealthCare Pharmaceuticals Inc. Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world’s leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women’s Healthcare. The company’s aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases. Bayer® and the Bayer Cross® are registered trademarks of Bayer. Intended for U.S. media only U.S. Media Contact: Marcy Funk, Communications, Bayer HealthCare Telephone: (862) 404-5385 E-Mail: Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.


News Release Intended for U.S. Media Only


If approved by the FDA, riociguat will be the first treatment for inoperable CTEPH or persistent/recurrent CTEPH after surgery and a new treatment for PAH

Whippany, N.J., August 6, 2013– Bayer HealthCare today announced that the U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal Drugs Advisory Committee recommended approval for investigational riociguat, proposed trade name Adempas™, in two forms of pulmonary hypertension. The Committee voted 11 to 0 that riociguat should be approved for the treatment of pulmonary arterial hypertension [PAH] of WHO1 Group 1. The Committee also voted 11 to 0 that riociguat should be approved for the treatment of chronic thromboembolic pulmonary hypertension (CTEPH) of WHO Group 4. In February 2013, Bayer submitted a new drug application for riociguat in two indications: (i) the treatment of PAH (WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening; and (ii) the treatment of persistent/recurrent CTEPH (WHO Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class. “We appreciate the Committtee’s discussion today around the safe and appropriate use of riociguat and are pleased with the outcome of the votes,” said Pamela A. Cyrus, MD, Vice President and Head of U.S. Medical Affairs, Bayer HealthCare Pharmaceuticals. “If approved, riociguat will offer a new treatment option for patients with PAH and will also provide the first approved non-surgical treatment option for CTEPH patients who are inoperable or who have recurrent or persistent disease. We look forward to continued dialogue with the FDA in order to make riociguat available to patients.”

1 World Health Organization

PAH and CTEPH are both life-threatening forms of pulmonary hypertension that cause significantly increased pressure in the pulmonary arteries. Riociguat is an investigational, oral medication for the treatment of adult patients with PAH or inoperable or persistent/recurrent CTEPH. If approved by the FDA later this year, it would create a new class of therapy available in the U.S. PH is associated with endothelial dysfunction, impaired synthesis of nitric oxide (NO) and insufficient stimulation of soluble guanylate cyclase (sGC). Riociguat stimulates sGC independent of NO and increases the sensitivity of sGC to NO. Data presented at today’s advisory committee meeting included results from the global Phase 3 clinical program, which enrolled 704 patients across two Phase 3 studies. Both studies met their primary endpoint by demonstrating a statistically significant improvement in the six-minute walk test (6MWT), after 16 and 12 weeks respectively. Riociguat was also associated with improvements across multiple, relevant, secondary endpoints in the studies. The most common treatment-emergent adverse events with riociguat were headache, dizziness, dsypesia, peripheral edema, nausea, diarrhea and vomiting. The advisory committee’s vote will be taken into consideration by the FDA when making its decision on the approvability of Bayer’s NDA for riociguat, which was submitted in February 2013. After acceptance of the NDA, the FDA granted riociguat priority review designation, which is given to drugs that have the potential to offer significant improvement in treatment or provide a treatment option where no adequate therapy exists. About Pulmonary Arterial Hypertension (PAH) In PAH, a rare and life-threatening disease, the blood pressure in the pulmonary arteries (the arteries that take de-oxygenated blood to the lungs from the heart) is significantly increased. PAH is characterized by morphological changes to the endothelium of the arteries of the lungs causing remodeling of the tissue, vasoconstriction and thrombosis-in-situ. As a result of these changes, the blood vessels in the lungs are narrowed, making it difficult for the heart to pump blood through to the lungs. In most cases, PAH has no known cause and, in some cases, it can be inherited. About Chronic Thromboembolic Pulmonary Hypertension (CTEPH) CTEPH is also a rare and life-threatening disease in which it is believed that thromboembolic occlusion (organized blood clots) of pulmonary vessels gradually lead to an increased pressure in the pulmonary arteries, resulting in an overload of the right heart. CTEPH may evolve after prior episodes of acute pulmonary embolism, but the pathogenesis is not yet completely understood. The standard treatment for CTEPH is pulmonary endarterectomy (PEA), a surgical procedure in which the blood vessels of the lungs are cleared of clot and scar material. However, CTEPH is inoperable in an estimated 20 to 40 percent of patients, and, in some cases, the disease persists or reoccurs after surgery. About Bayer HealthCare Pharmaceuticals Inc.Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world’s leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women’s Healthcare. The company’s aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases. Bayer® and the Bayer Cross® are registered trademarks of Bayer.

Intended for U.S. media only Media Contact: Marcy Funk, Communications, Bayer HealthCare Telephone: (862) 404-5385 E-Mail:

Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

Posted in About Us, Diseases, Featured | 1 Comment

Pillow Fight: Sleep and Chronic Illness (Part 2)

Posted on by Charlie

Trouble tossing and turning? Experts offer tips for getting better sleep, plus an overview of sleep disorders and sleep testing, from A to Zzzzzzzzzzz.

Timothy Morgenthaler, M.D., president of the American Academy of Sleep Medicine (AASM), and professor of medicine at Mayo Clinic, discusses sleep and chronic illnesses, including pulmonary hypertension, Huntington’s disease, and narcolepsy, and gives tips on regulating sleep, and testing for sleep disorders.

Click here to read part 1.

Sleep and Pulmonary Hypertension

The current American College of Chest Physicians (ACCP) guidelines for evaluation and management of pulmonary hypertension strongly recommend an evaluation for sleep-disordered breathing, in particular obstructive sleep apnea syndrome, both because it can be contributory, and in severe cases, causative of pulmonary hypertension.

Photo by Retroklips

Photo by Retroklips

Recent literature shows a very high percentage of patients who have World Health Organization (WHO) Class 1 pulmonary hypertension have either obstructive or central sleep apnea. Furthermore, there’s really a growing body of evidence that shows that when you treat sleep apnea using positive airway pressure, not only does it treat the sleep consequences of having these sleep disorders, it also lowers the pulmonary artery pressure.

What I think the new information showing a high prevalence of central sleep apnea means for patients who have significant pulmonary hypertension, is that they will be best served to be evaluated by a board-certified sleep specialist, who has significant expertise in diagnosing sleep-disordered breathing and in management with the different devices that we can use to manage sleep apnea. People could find such specialists at an AASM-accredited sleep center, or they can look on, and locate sleep experts close to them.

Pulmonary hypertension patients can have other sleep diseases, but they do tend to have a high prevalence of fatigue as a symptom. Fatigue management can also sometimes be helped by a sleep specialist.

Sleep and Huntington’s Disease

Huntington’s disease is less common than Parkinson’s or Alzheimer’s or Lewy body, all of which are degenerative central nervous system (CNS) diseases. What’s come to light in recent times is how significant sleep concerns are in Huntington’s patients. They basically weren’t asked in the past. Now that they’re asked, they tell.

A significant sleep problem is that their circadian rhythm gets quite upset, such that their wake and sleep patterns get very off-center from what the rest of their community wants to obey.

Given that, in some of the other diseases, like in Alzheimer’s disease, sleep issues are actually one of the primary reasons for institutionalization, it’s probably very important that we pay special attention to patients’ sleep complaints in Huntington’s disease as well.

Sleep and Narcolepsy

Narcolepsy is probably best managed by sleep specialists who have gone through fellowship training to learn how to manage narcolepsy and other primary CNS hypersomnias.

It’s a very interesting hypersomnia that has increasingly known cause. At least at the anatomical level, we know about the hypocretin system and how that seems to be not functioning well in patients with narcolepsy.

We’re still at a point where narcolepsy diagnoses rely on well-done sleep studies. We have quite a large referral for narcolepsy here at the Mayo Clinic Center for Sleep Medicine. One of the real problems is that very often people coming to us have had less than optimal diagnostic evaluations.

Our current tools for diagnosing narcolepsy are sleep studies, and they involve, first of all, obtaining a very accurate history of peoples’ actual sleep patterns and how they are pursuing those, followed by an overnight polysomnogram, which is our most accurate tool for knowing exactly how that person sleeps and what sleep stages they have, followed by multiple sleep latency testing.

A good nighttime ritual is helpful.

Very often, when patients are referred to us for management of narcolepsy or reevaluation, because something hasn’t quite worked out right, we find that the evaluation has been done inappropriately, or not very optimally.

Either it was not clear what medications they were on when they were having the test, and that turns out to be very important, or it was not objectively documented what their sleep schedule was prior to the testing. There are many things that can go wrong, and in order to really orchestrate getting that test sequence done correctly, it takes work.

I really think that, wherever possible, that type of diagnostic evaluation should be done at an accredited center that’s going to adhere to the guidelines and should be done very carefully. Because you are rendering a service, you hope, to a patient, and it’s one that’s going to give them a diagnosis, and it’s a chronic illness that we don’t have a cure for.

You’re really committing to a lifelong management of an illness, and we should take great care to make sure we have the right diagnosis, so that our treatments make sense.

Sleep and Alpha-1 Antitrypsin Deficiency

Patients with advanced emphysema [such as the genetic or inherited emphysema caused by alpha-1 antitrypsin deficiency] certainly have a poor quality of sleep. They have a higher prevalence of, not only breathing problems, but also tend to have much more fragmented sleep.

How much of that is due to anxiety, depression, and discomfort, I don’t think is well known, but even there, there are guidelines that can help patients with such severe emphysema sleep better.

Photo by Retroklips

Photo by Retroklips
Sleep, Childhood Epilepsies, and Pediatric Narcolepsy

Althea Robinson-Shelton, M.D., assistant professor, Neurology-Sleep Division, Vanderbilt University, discusses the relationship of sleep to childhood epilepsies and pediatric narcolepsy, testing for pediatric sleep disorders, and the importance of pediatric sleep hygiene and ritual.

Pediatric Sleep Hygiene and Ritual

It is important to establish a consistent sleep ritual and sleep environment for children. Starting a wind-down time before bed is a good idea. This is a time, 45 minutes to an hour before bed, when all electronics are turned off, and non-stimulating activities, such as reading, drawing or writing are done.

‘What is bedtime? Is there a TV in the room? Are there electronics in the room?’ are just a few important sleep hygiene questions.

Sleep and Pediatric Narcolepsy

Narcolepsy may initially present solely with excessive daytime sleepiness. If cataplexy (loss of body tone with strong emotion) is not initially present, it may develop later. We now know that the [average] lag time from onset of symptoms to diagnosis is about eight years.

Many narcolepsy patients’ symptoms started in adolescence, but they were labeled as lazy, or [told] they weren’t trying hard enough. In reality, they are trying their best to stay awake. They’re not intentionally sleeping in class or sleeping all day. It’s really difficult for them to stay awake.

If your child is getting sufficient sleep, meaning they’re going to bed in a timely manner, falling asleep in a timely manner (within 30 minutes), and sleeping through the night, but they’re still having excessive daytime sleepiness, there is concern for a possible sleep disorder. It may not be narcolepsy, but narcolepsy is on the differential diagnosis.

Sleep and Childhood Epilepsies

Sleep and epilepsy are intertwined. Chronic sleep deprivation can lower seizure threshold, which in turn can increase seizure frequency. This phenomenon can become a perpetual cycle, where chronic sleep deprivation can lead to greater seizure frequency, and an increase in seizure frequency causes sleep disruption.

When a child comes in with a sleep-maintenance insomnia (they fall asleep without issue, but have difficulty maintaining sleep), there are potentially a number of causes, but if they have a history of epilepsy, nocturnal seizures may be the cause. Other disorders that can cause disruption in sleep, such as obstructive sleep apnea (OSA) and periodic limb movement disorder (PLMD), must also be considered.

Parents often don’t suspect children of having OSA, or [they think] that snoring is normal. Snoring, especially when it is in association with respiratory pauses, gasping and/or choking is concerning for OSA. Untreated OSA causes sleep deprivation, which can increase seizure frequency.

If a parent is concerned about a possible sleep disorder in their child, the first step is evaluation by a sleep specialist. Based on the clinical history and exam, a polysomnogram (PSG) may be recommended.

If narcolepsy is in the differential diagnosis, a PSG to exclude disorders that fragment sleep (for example, OSA or PLMD) will be ordered along with a multiple sleep latency test (MSLT).

Evaluating and treating sleep disorders in childhood improves quality of life and may improve seizure control in patients with epilepsy.

Sleep Resources

A sleep health information resource by the American Academy of Sleep Medicine with information on AASM-accredited sleep centers, healthy sleep awareness, sleep disorders, disease management, news, a video archive and a sleep product guide.

The National Center for Complementary and Alternative Medicine website features information about sleep disorders and complementary health approaches, including relaxation techniques, yoga and meditation. Talk to your health care providers before trying a complementary health approach for sleep problems.

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Pillow Fight: Sleep and Chronic Illness (Part 1)

Posted on by Charlie

Trouble tossing and turning? Experts offer tips for getting better sleep, plus an overview of sleep disorders and sleep testing, from A to Zzzzzzzzzzz.

Timothy Morgenthaler, M.D., president of the American Academy of Sleep Medicine (AASM), and professor of medicine at Mayo Clinic, discusses sleep and chronic illnesses, including pulmonary hypertension, Huntington’s disease, and narcolepsy, and gives tips on regulating sleep, and testing for sleep disorders.

Photo by Retroklips

Photo by Retroklips
Sleep Medicine Trends and Research

There’s growing public awareness of the importance of sleep. Sufficient sleep is definitely considered one of three pillars of a healthy lifestyle, as important as good nutrition and regular exercise.

Public awareness is also growing of just how common sleep illness is. More than 70 million Americans suffer from a sleep problem, and over two-thirds of them have chronic sleep disorders that can actually affect their overall health. To learn more about that, is a very good site with further links to good sleep education.

Along with this awareness, there’s more and more research about how important our circadian biology is in our performance and in our quality of life, and in things like regulation of weight.

I think most people are aware of our obesity epidemic, and it’s probably only been in the last few years that people are really beginning to connect the dots, that part of the obesity epidemic is the insufficient sleep epidemic. We know that there’s a tight linkage there.

Photo by Retroklips

Photo by Retroklips

Obstructive sleep apnea is a serious medical illness, affecting as much as 20 percent of the Medicare-age population. Along with already existing effective treatments, new technologies are being developed to treat sleep apnea. These advancements in treatment techniques will offer more and perhaps better ways to manage this illness.

Research in the pathophysiology of narcolepsy is also leading to the development of new therapies for sleep. There are advances in circadian biology, treatment of sleep-disordered breathing, diagnostic capabilities, and the neurobiology of sleep.

Patients with chronic illnesses should really value their sleep. They should strive hard to achieve adequate sleep, first making sure they’re following healthy sleep habits.

If they are not rested, despite their best efforts, they should seek help from their physicians, and in many cases, they would benefit from referrals to a sleep specialist at an American Academy of Sleep Medicine (AASM)-accredited center. We have over eight-thousand sleep specialists in the United States. Sleep is important, and we can often help. We have very good capabilities to diagnose and treat sleep.

Regulating Sleep

For people both with and without chronic illness, many of the same things apply in regulating sleep.

People who exercise regularly tend to sleep better.
Seeking Treatment

If even after following good sleep hygiene, you have sleep apnea, either obstructive or central, you’re still not going to sleep as well as you might, and you really should seek treatment for your sleep condition.

Also, with some of these disorders, people tend to have more chronic anemia, and anemia can be associated with restless leg syndrome, which can also cause problems for sleep. That’s another sleep condition that can be appropriately diagnosed and treated, usually very effectively.

Sleep Misconceptions and Sleep Deprivation

The sleep misconception we hear the most that is the least true is: ‘I can get by just fine with only five or six hours, and then I make it up on the weekend.’

What most studies show about how sleep deficit affects us, is that it’s very much like alcohol, like being out with a friend who has had one drink too many, but they don’t think they have. They think they’re just fine. But you know them well, and you’re watching them, and their mental acuity, judgment, and coordination aren’t so good.

Sleep deprivation is a lot like that. Studies show that we’re very poor at really detecting how our lack of sleep is affecting our performance. We don’t actually feel as sleepy as we are. We don’t realize that we’re not thinking as well as we should. What study after study shows, is that you really do need about seven hours or more of good quality sleep to be at your best.

When you get less than that, you tend to crave the wrong foods, and it tends to be associated with the development of medical illnesses. It tends to decrease your cognition, reaction time, coordination, mood, and judgment. People can become very convinced that they really don’t need that.

They’ll often say, ‘Well, I’ve read about people who are short sleepers.’

There are a few, very few people who can get by and not show detriments until they get quite a bit less sleep. But that’s very few people, and it would kind of be like suggesting, ‘I know people who can run the marathon in less than four hours, and so I can.’ That isn’t going to happen.

Sleep Facility and Sleep Specialist Accreditation

Board certification for sleep specialists is given by the same American Board of Medical Specialties that certifies critical care medicine, pulmonary medicine, and neurology. It’s a medical specialty, like any other.

Accreditation of a sleep center basically takes into account a review of the facilities. What is the physical plant of the place like? Is it safe for patients? Of the processes, are referral patterns, records, treatment pathways, and emergency procedures best practices?

There’s also a third leg that looks at the diagnostic and therapeutic procedures. Any facility that’s accredited by the American Academy of Sleep Medicine (AASM) must follow the standards that are laid out in our treatment guidelines and practice parameters.

When facilities are AASM accredited, they have to be able to demonstrate that they are already following the current practice parameters and treatment guidelines. We very tightly link accreditation with state-of-the-art practice

Finally, there’s an important emphasis on quality improvement, so that they’re monitoring quality and have active programs to improve their quality.

Diagnostic Testing

Different diagnostic tests that might be used at a sleep center would include actigraphy, which is a way to add to a sleep diary and learn more about the sleep patterns of a patient.

A polysomonogram (PSG) is typically an in-facility monitored test that measures brain waves, muscle tone, eye movements, oxygen saturation, EKG, efforts to breathe, and air flow, so it’s the most comprehensive test of sleep.

The multiple sleep latency test (MSLT)offers five nap opportunities during the day, and we measure how long it takes for a patient to fall asleep and what stages of sleep they fall into.

The maintenance of wakefulness test (MWT) is similar to the MSLT, but kind of the opposite instructions are provided to the patient: Please stay awake in a very boring environment, and we see how effective they are with that. There can be home sleep apnea tests that are applicable to many patients who might have sleep apnea.

There are blood tests that are sometimes useful in evaluating, for example, restless leg syndrome, or sometimes narcolepsy, or thyroid disease. We might look at blood tests to help us make those diagnostic assessments, and depending on the history and the presentation, we might look at other tests of cardiac and pulmonary function as well.

Medication and Sleep

In management of all chronic illnesses, there’s a component where you want to use lifestyle changes and non-pharmacologic approaches to the illness, and then sometimes it’s wise to add a pharmacologic component.

As a sleep specialist, we use medications often to help with sleep, but you might be surprised. [With] many patients who come to us for insomnia, we would prefer to use cognitive behavioral type of therapies, which are actually safer, at least as effective and don’t involve controlled medications. You have to really develop a comprehensive picture of patient needs and then pull out all the tools to serve those needs.

Click here to read part 2!

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To Your Health

Posted on by Charlie

CVC Senior Case Manager Lauren Ruiz explains open enrollment for health insurance, the different types of Medicare plans, and common health insurance terms.

Open Enrollment Details and Deadlines

Deadlines in health insurance are like your child’s first steps— you don’t want to miss them. One day could mean the difference between the effective date you want, enduring another month without insurance before your coverage starts, or losing the opportunity to enroll altogether.

Annual Open Enrollment Periods exist for almost all insurance types since the enactment of the Patient’s Rights and Affordable Care Act. Applications for Medicaid are still accepted all year long, but outside of that, some important dates to remember are:

It is possible during these enrollment periods to find good, comprehensive health coverage that doesn’t break the bank. It is also possible to make it through the entire insurance process with all of your hair still intact—you just need the right tools and preparation.

Most importantly, know your options. Try not to completely dismiss the idea of a specific policy type simply because you aren’t familiar with it. Everyone has different health needs, and what works for one person may not be the best coverage for someone else. The perfect example of this can be seen through the variety of Medicare plans available.

Medicare Plans

If you’re not familiar with Medicare, there are several different types of coverage a member can purchase – Original Medicare, a Medicare Supplemental Plan (Medigap), a Medicare Advantage Plan, and a Medicare Prescription Drug Plan.

Original Medicare is the coverage you automatically receive when you turn 65 years of age, or have been approved for Social Security Disability benefits for 29 months. Part A, hospital coverage, is provided at no cost to those who qualify and Part B, medical coverage, is available for a monthly premium. With Original Medicare, members typically pay 20 percent of the cost of most services, and prescription coverage is not included.

Medicare Supplemental Plans do exactly as their name indicates; they supplement the costs left behind by Medicare. The 20 percent member responsibility is covered completely by a Supplemental Plan. Certain plans—denoted by specific letters like Plan “F”—even cover additional costs like deductibles. These policies tend to have a higher monthly premium than other Medicare options, however, so while they offer comprehensive coverage, they may not be realistic or necessary for everyone.

Medicare Prescription Drug Plans, again, cover what their name suggests—prescription drugs. Typically, plans with great drug coverage are available at monthly costs under $50, and most plans follow a standard co-payment structure. If you have only Original Medicare, or Original Medicare and a Supplemental Plan, you will probably want to purchase a Prescription Plan as well.

Finally, a Medicare Advantage Plan covers hospital costs, medical costs, and prescription costs, all bundled together, under one policy. The “advantage” to these plans is that the hospital and medical coverage has an associated out-of-pocket maximum, meaning once that amount is met, the member will be covered at 100 percent for those services. Different kinds of Medicare Advantage plans are available—HMO, PPO, HMO-POS and more—and each has different rules to follow, so educating yourself about common insurance jargon can help make your insurance journey easier.

Common Health Insurance Terms

Understanding common insurance terminology will give you the ability to make the most informed decision and will allow you to consider your coverage from every angle, so you don’t miss any important benefits. Here are some terms every insurance policy holder should know:

• Summary of Benefits Coverage (SBC) – A document that details all the specific benefits and costs of a plan. It will detail what services are covered, what is not covered, and how much each will cost.

• Deductible – The amount the member pays before coverage kicks in. All costs are paid 100 percent out-of-pocket until the deductible amount has been met. Plans may have separate health deductibles and prescription drug deductibles.

Co-payment – A flat amount the member is charged. Co-payments typically vary by service, so you may have one flat charge to visit your primary care physician, another to visit a specialist, and another to visit an emergency room.

• Co-insurance – The member is responsible for a percentage of the full cost of a service or medication. For example, a 20 percent co-insurance on a $400 lab test means the member will pay $80.

• Out-of-Pocket Maximum (OOPmax) – The most a member will pay in co-payments and co-insurances during the year, and then the plan will cover all charges at 100 percent. There may be certain charges that do not apply to the out-of-pocket maximum. If the plan also has a deductible, check to see if it is included in the out-of-pocket maximum amount.

• Benefit Maximum – A limit on the number of visits or total amount a plan will cover during the year or over the lifetime with the plan. Once the benefit maximum is met, the member will pay 100 percent of the costs for those services.

• Out-of-Network – Services received from a physician or medical facility that is not contracted with your plan. You will pay more for out-of-network services, or your plan may not cover any of the costs. Most out-of-network costs will also not apply toward the deductible or OOPmax.

• Prior Authorization (PA) – An approval from the insurance policy for a prescription drug before the plan will pay anything for the drug. Your prescribing doctor can submit a PA to the insurance company.

• Quantity Limit (QL) – The maximum amount of a prescription drug allowed within a 30-day period. If you are prescribed more than the maximum amount, the plan will not cover the costs for the additional medication, unless the prescribing doctor submits a request to increase the QL, and it is approved.

• Step Therapy (ST) – A restriction placed on a medication by the plan that requires the member to try alternate, cheaper options of a prescription drug before the plan will pay for it. If you have already tried the other drugs, and they didn’t work, or if you are on other medications that will negatively interact with the proposed alternatives, your prescribing doctor can submit a request to bypass the ST requirement.

• Not on Formulary – The plan does not cover the prescription drug and will not pay for it. Your prescribing physician can submit a Formulary Exemption request to the insurance. If it is approved, the plan will help pay for the medication.

• Explanation of Benefits (EOB) – A document the member receives from the insurance plan whenever a claim is submitted to the insurance company for payment. The EOB is not a bill, but it details how much the plan paid for a service and what the member’s cost will be. Always check the EOB to make sure you are being charged the right amount. Compare the SBC with the EOB if you are unsure, and call the insurance company if you have any questions.

• Health Maintenance Organization (HMO) – A type of insurance policy classified by its strict network of physicians and medical facilities. A member must stay In-Network to receive coverage and may have to get a referral to see a specialist doctor. Costs are usually lower in these policies, as long as your doctors are In-Network.

• Preferred Provider Organization (PPO) – A type of insurance policy with no restrictions on what doctor you can see or which medical facility you can go to. There is still an In-Network list which costs less to visit, but the plan will help pay for covered services, regardless of the doctor.

• Point-of-Service (POS) – A type of insurance policy that has a specific In-Network list, but will help pay for covered services submitted by any doctor, as long as that doctor will accept the rates charged by the insurance plan. If you want to visit a physician or medical facility not listed in the plan’s network, call the doctor and ask if they will accept the plan.

Record Keeping, Your Needs, and Coverage

Knowing your options and educating yourself gives you the tools—now you just need to be prepared to begin the insurance process. Keep a record of all your important insurance documents, including Letters of Creditable Coverage, Notices of Plan Termination, EOB’s, and any additional grievances, requests, or appeals that may have been filed with an insurance company. These may be vital in securing a Special Enrollment Period that falls outside of the open enrollment periods first mentioned, or in disputing a charge with your previous or existing insurance plan.

Make a list of your insurance needs and what you can afford. If you receive Durable Medical Equipment (DME) for any reason, make sure you enroll in a plan that pays for DME. Include all the services and prescription drugs you know you need, as well as all additional expected services in your list. No one can predict the future, but talk to your doctors and ask if they foresee any specific upcoming procedures or prescriptions, so you can check the insurance plan for coverage of those as well.

If you are thinking about switching insurance plans during an open enrollment period, or are newly eligible for coverage, call Caring Voice for assistance. We can discuss your options and make sure you have the tools and preparation to find the best coverage for your needs.

January 1 – March 31
October 15 – December 7
November 15 – February 15
Medicare General Enrollment Period to elect Original Medicare if your Initial Enrollment Period has already passed
Medicare Prescription Drug Plan Open Enrollment Period to add new coverage or change your existing Prescription Drug Plan or Medicare Advantage Plan
Private Health Insurance Open Enrollment Period to elect coverage through a Federal or State Marketplace or directly from a private insurance carrier
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In Your Words: Debora Camp and Huntington’s Disease

Posted on by Charlie

Debora Camp was adopted and didn’t grow up knowing that Huntington’s disease was part of her genetic heritage. Her husband, Stephen Camp, describes how he and Debora deal with the illness that took them by surprise.

Deb and Stephen Camp, Buca di Beppo restaurant, Pineville, North Carolina.

Deb and Stephen Camp, Buca di Beppo restaurant, Pineville, North Carolina.

Deb and I grew up in the same church, so our memories of each other go back to childhood. We attended different high schools, but later attended the same college. I suppose we were opposites in many ways, Deb, the extrovert, and me, the shy one. We married in 1973 and have three children and five grandchildren.

Deb’s Huntington’s disease came as a complete surprise to us, since Deb was adopted. The diagnosis came in 2009 when her family doctor was concerned about odd movements Deb showed during a medical appointment. In retrospect, we can recognize other earlier symptoms, but that symptom is what resulted in a referral to a neurologist.

Deb and I have always found moments to laugh at our predicaments, and as strange as it may seem, we even found moments to laugh when we got the shocking news of her diagnosis. Deb’s adoptive mom had Parkinson’s, and while we waited for the neurologist’s diagnosis, she wondered whether that might be her fate as well.

When she got the Huntington’s diagnosis, she came home and said to me, “The good news is I don’t have Parkinson’s or Alzheimer’s. The bad news is, I have both.” Deb is a very independent and determined person. She insists that she will live as fully as possible for as long as she lives, and humor will always be part of living.

Our advice to anyone newly diagnosed would be to act as proactively as possible. A recent incident was shocking, but was the impetus to prepare for and prevent future similar incidents.

Deb Camp at the Lazy 5 Ranch, Mooresville, North Carolina.

Deb Camp at the Lazy 5 Ranch, Mooresville, North Carolina.

Deb was traveling with our daughter and three grandchildren recently when they stopped for a bathroom break at a donut shop. Deb accompanied the children to the restroom, while our daughter saw to other tasks.

Suddenly, three police cars pulled into the parking lot and blocked the exit. A policeman approached Deb. She was distributing juice boxes to the children as they fastened themselves into their backseat restraints. He began to question her and ordered her to get out of the car.

He then pulled her hands behind her back, presumably to handcuff her. My daughter ran around the car yelling, “She has Huntington’s.” The policeman stopped and apologized and asked if he could be helpful. Apparently, someone in the donut shop had thought Deb was drunk and called the police.

Deb explained to the grandchildren through her tears that the police thought she was sick. She went home and ordered a medical bracelet that indicates she has Huntington’s and she wears that whenever she leaves the house. We’ve also discussed Deb’s Huntington’s with our local police department. HDSA has a very helpful caregiver guide designed for police departments that can be found at the HDSA website. (See below.)

Huntington’s Disease Information for Caregivers and Law Enforcement

The HDSA Caregiver Guide, providing suggestions and advice for potential law enforcement and other emergency services personnel encounters, can be found by clicking this link

Call HDSA at 800-345-HDSA for more information.

Deb and I both spent much of our careers as mental health counselors on college campuses. One might think that helps us deal more effectively with difficult circumstances in our lives. Well, Huntington’s has its surprises, and any day can bring a new challenge.

Deb didn’t grow up knowing that Huntington’s was a part of her genetic heritage. She never saw an older relative and her family cope with it as a fact of life. Every difficulty is new to us.

We are now strategizing to make sure Deb has regular check-ins from friends or potential caregivers, since I am often away from home. Deb and I are both concerned about the potential for a fall, and we’re evaluating our home for ways to reduce the risk. We are working with Deb’s doctor to be sure she is getting the right medication to help manage her mood, sleep, and energy.

Deb strives to be as active as possible. She has always loved children, the outdoors, animals, and singing. She is active in Animatera, a southwestern New Hampshire women’s singing group.

We live in a rural setting and have always been hobby farmers. The usual routines of a hobby farm are now much more difficult, but we manage with the help of neighbors.

Deb is retired, but I continue to work in higher education. I am a dean in Student Affairs at a community college. I love my work, but can never stop worrying about Deb and even our children and grandchildren who are at risk.

Spouses of persons with Huntington’s need to care for themselves too. Not long ago, I was diagnosed with Parsonage-Turner syndrome, an autoimmune disorder that is quite rare. In my case, it resulted in paralysis of my diaphragm.

Since I’m healthy otherwise, I was able to adjust easily by using the other torso muscles involved in breathing. I’m convinced, however, that my illness is partly a stress response to Deb’s illness. I’ve determined that loving Deb also requires that I care for myself and seek help when I need it.

Huntington’s disease is a challenge that researchers are taking on, and Deb expects to enter some medical trials this summer. My friends and brothers and sisters have been very supportive. These days, I am encouraging all of them to learn more about Huntington’s disease and to help in any way they can to work toward a cure.

Posted in Diseases, Featured, Media Center, Uncategorized | Leave a comment

New Office Hours

Posted on by Charlie

Starting Tuesday, September 2, 2014, CVC will have new office hours, Monday–Friday, 9 a.m.–6 p.m. EST.

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Close Up: Nicole Paulsen, CVC Events Coordinator

Posted on by Charlie

Nicole Paulsen, CVC Events Coordinator.

Nicole Paulsen, CVC Events Coordinator.

I always wanted to work for a nonprofit doing event planning. My mom ran a daycare when my three older brothers and I were younger. We had the best childhood growing up. In the summers, my mom planned these elaborate, fun, themed events for all of the kids in our neighborhood. We would have “Backyard Circus Day,” “Summer Olympic Challenges,” and massive scavenger hunts on a weekly basis.

My mom now works for our county parks and recreation department, where she plans community and youth programs. Throughout high school and college, I found myself working with my mom on various youth events and summer camps and truly loved it.

I love working at CVC because I get to do fun things, not only for the patients, but also for the employees. I love to plan activities that get people excited and give them something to look forward to, like monthly events for CVC staff. I also make travel arrangements for our staff to attend many of the annual patient conferences and education days.

Speaking to patients at the conferences, I’ve learned that travel with chronic conditions can be stressful. For them, making travel arrangements is a bit more involved than for most people. They have to let the airline know in advance if they’re traveling with a POC or a CPAP machine and make sure it’s FAA-approved. Each airline has a different procedure.

Some of our patients need wheelchair assistance in the airports, accessible hotel rooms, and hotel rooms with refrigerators for their medication. Narcolepsy patients with cataplexy need to have tubs so they don’t fall in the shower and hit their heads. Many of our patients have to take their medication, eat, and sleep at certain times when they’re traveling. Some have to have infusions on a weekly basis and plan their travel around that. Patients have to have prescriptions for their machines and medications on them at all times when they travel.

The relationships they form at patient education days and annual conferences can be even more beneficial than a typical vacation. They meet people with rare and chronic diseases who are exactly like them. It can be very comforting. When they’re first diagnosed, it can be very scary. They often tell me that their doctors don’t know anything about the disease. Going to education days and conferences can be quite meaningful to them.

What I think is most important for the newly diagnosed to know, is that, when they call here, they’re not going to be sent to a typical call center. Everyone here at CVC genuinely cares about what our patients are going through and will do their absolute best to help them.

CVC receives so many thank-you notes on a daily basis. Our patients take the time to pick the cards and write heartfelt thank-you notes. Reading them, you truly understand how much of a difference we have made with their quality of life, and that is very powerful.

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Legal Corner: Open Roads

Posted on by Charlie

CVC Patient Advocate Stephanie Posuniak explains the rights of travelers with special needs, both in the U.S. and abroad.

Research suggests that the act of planning and anticipating a vacation boosts happiness, and that a very relaxed vacation boosts mood upon return. If you’re planning a vacation, this article will help you prepare for your journey and understand the laws that protect you and help make travel barrier-free.

Domestic Travel

The Air Carrier Access Act (ACAA) prohibits air carriers flying to and from the U.S., its territories, possessions, and commonwealths from discriminating against passengers on the basis of physical or mental disability. The Department of Transportation (DOT) has issued a rule providing standards of service which air carriers are expected to provide disabled individuals.

Manor sleeping car, Toronto-Vancouver route, Via Rail, Canada. Photo by Bianca Courtemanche.

Manor sleeping car, Toronto-Vancouver route, Via Rail, Canada. Photo by Bianca Courtemanche.

The DOT also provides a toll-free hotline to provide consumers with general information about the rights of air travelers with disabilities, respond to requests for printed consumer information, and help air travelers with time-sensitive disability-related issues. The hotline’s hours are 9 a.m. to 5 p.m. EST, Monday through Friday, except federal holidays. Call 1-800-778-4838 (voice) or 1-800-455-9880 (TTY) for assistance.

Requesting information from your airline prior to flight is also a great idea; for example, how will passengers embark, what storage facilities will be available, and what type of lavatory will be on the plane? The Americans with Disabilities Act (ADA) requires that carriers provide this information.

Generally, you’re not required to give advance notice to the air carrier that you have a disability; however, it may be a good idea to do this so the attendants will be ready to assist. There are some situations in which you must give advance notice to the air carrier. For example, when using your FAA-approved oxygen concentrator in-flight, you may need to present a statement from your physician confirming that you can safely undertake the flight.

If you elect to have an attendant on board, remember that the attendant is there for emergency evacuations and is not there for personal services like assisting with eating or accessing the lavatory. The carrier cannot impose a charge for the transportation of a requisite safety assistant.

Cruise Lines and Railways

Domestic cruise lines must also comply with the ADA by taking steps to accommodate passengers with disabilities. Under the ADA, a cruise line must not:

The ADA also applies to domestic passenger railways. Under the ADA, railcars must contain:

Sleeping compartments on railways must allow a person using a wheelchair or mobility aid to enter and maneuver within the compartment.

Service Animals
You don’t need to provide certification for your service animal in the airport as long as you provide “credible verbal assurances” that the animal is a service animal. With that in mind, bring documentation just in case.

The ADA defines “service animals” as those that work or complete a task “directly related to the person’s disability.” Thus, if the animal is solely for comfort or emotional support unrelated to a disability, the animal does not qualify as a “service animal” under the ADA.

When going through airport security, let the security officer know that the animal is a service animal, which means the officer cannot separate you two. While going through the metal detector, you may choose to have your animal go before or alongside you, whichever is better. The officer is trained in how to conduct searches of individuals with service animals and is subject to specific rules. The rules state that the officer cannot intentionally touch your animal without your permission. But you have a duty to assist with the inspection by controlling the animal. You may keep your service animal with you in-flight.

If traveling within the United States, the ADA applies to hotels, motels, inns, and other places of lodging built later than January 26, 1993. The ADA requires these structures accommodate individuals with disabilities and includes detailed design requirements. Dwellings subject to the ADA must also provide van-accessible parking spaces depending on the total number of rooms. There must also be at least one accessible route for those using a wheelchair or other assistive devices to approach and enter the building. Depending on the number of rooms, the hotel must also provide roll-in showers.

Health Insurance
Will your health insurance cover you while traveling? If traveling within the United States, regular Medicare rules apply for Part A and B coverage (“original Medicare”). If you have a different type of health insurance than Medicare, check your policy for what’s covered.

If you have an Advantage Plan, the rules differ depending on how long you travel outside the plan’s service area. If you travel outside the plan’s service area continuously for more than six months, most plans will automatically dis-enroll you and enroll you in Original Medicare, if, in the meantime, you do not choose another Advantage Plan. If outside the service area for less than six months, whether the plan will cover you depends on the type of plan you have (PPO vs HMO). Generally, HMOs do not cover services from providers outside the plan’s network.

For Part D, check with your plan, the pharmacy, or call 1-800-MEDICARE for information on whether your preferred pharmacy is on your Part D Plan’s preferred list. The same applies for Medigap policies: see your Medigap policy for more information.

International Travel


Under the ACAA, both domestic and foreign carriers flying to or from the U.S. must:

Cruise Lines and Railways
Cruise ships that dock at U.S. ports must comply with the ADA, and the U.S. ports must also be ADA-compliant. For new port facilities, the operator must ensure that individuals with disabilities, including those using a wheelchair, can use the facilities. Existing ports may have to remove architectural barriers, if possible, and if not, provide reasonable alternative accommodations.

European railways may not be wheelchair-accessible and are not subject to the same United States standards. Contact the local embassy to determine what services and accommodations they may extend.

Waikiki, Oahu, Hawaii. Photo © Hawaii Tourism Authority (HTA)/Tor Johnson.

Service Animals
Foreign carriers are not subject to the ADA and may only accept service dogs. Check with your destination’s embassy. You will also want to carry documentation showing that your animal is a service animal and not a pet. It’s also a good idea to learn the foreign country’s laws and regulations pertaining to leashing or muzzling and your liability if your service animal were to bite.

Traveling with a service animal also requires consideration of cultural and environmental issues. What are the country’s cultural customs regarding your animal? How will the public behave toward your animal? Prepare your service animal by exposing him to the climate, crowds or environment he will experience.

Also be mindful of your animal’s health. What changes in diet, grooming, and personal care will the animal experience? Determine whether the drinking water will be safe and to what parasites or viruses he may be exposed. You’ll also want to get his vaccinations up-to-date and carry a record of that.

Depending on your destination, you may need to make arrangements with the target hotel to assure accessibility.

Health Insurance
The Medicare rules become more complex in the international arena. “Outside the United States” means anywhere other than the 50 states, District of Columbia, Puerto Rico, U.S. Virgin Islands, Guam, American Samoa, and the Northern Mariana Islands. Generally, Part A (which predominantly covers in-patient care and home health services) does not cover you while overseas, except in limited situations. For example, if you experience a medical emergency and the foreign hospital is closer than the nearest U.S. hospital, Part A may cover you.

Part B (which covers services such as out-patient doctor visits) does not extend overseas unless in specific situations. For example, Part B may cover medically-necessary services on board a ship within the territorial waters adjoining the land areas of the United States if the ship is six hours or less in distance away from a U.S. port.

If you have a Part D Plan, remember that drugs are not covered if bought outside the United States. If you have a Medicare Advantage or Medigap Plan, check with your plan to see whether you are covered abroad. If you find that you are uninsured or underinsured overseas, you can buy a supplemental health insurance policy specifically for traveling.

Prescription Medications
When bringing medications into a different country, be familiar with the country’s laws on what drugs are legal and what documentation is required. If unsure, contact the embassy.

I hope you notice the mood-boosting effects when planning your next vacation! If you start to feel overwhelmed, remember, as the Chinese philosopher Lao-tzu said, “A journey of a thousand miles begins from beneath your feet.”

Posted in Diseases, Featured, Media Center | Leave a comment

Learning to Live with Pulmonary Hypertension

Posted on by Charlie

48-year-old Mari Jackson had worked since the age of 12. But last year, a diagnosis of pulmonary arterial hypertension forced her to slow down and approach life differently.

Mari Jackson and her husband, William.

Mari Jackson and her husband, William. Photo by Anthony Nesossis

I got diagnosed with pulmonary hypertension last March. The symptoms started in February of last year, right around my husband’s birthday. It was like something out of the blue.

We had gone to dinner to celebrate his birthday and came home, and I said, “I’m going to get ready for work.”

I went upstairs to take a shower, and when I got off the bed to walk towards the bathroom, I just blacked out. When I woke up, I was on the floor, and I was face down by the sink.

I called my husband, and he and my son came running up the stairs, and my husband said, “What are you doing on the floor?”

I said, “I have no idea. I just blacked out.” My body had just shut down. I was so embarrassed.

But I didn’t pay it any attention. I worked for a mortgage company. Believe it or not, Monday through Friday, I drove every day to work—44 miles round trip. I worked anywhere from 10 to 12 hours a day, and I worked weekends and overtime.

I had an episode every weekend. I would pass out. I actually had a seizure one night. My husband noticed that every time I would go upstairs, I would get really, really light-headed and either fall backwards or lose my balance.

So, one night he came upstairs with me and said, “Let me just help you into the room.” I was using the bathroom, and I was sliding off the seat. I had a seizure, and he was right there to catch me, and I bit my tongue, and it was so weird because I had never had that happen.

He took me to the ER, and they ran a lot of tests. They said, “We don’t see anything, but we do see that you have pneumonia.” So, they gave me antibiotics for that, and I went home.

The first Saturday in March, I went to work. I got upset about something, and the whole left side of my arm turned ice cold. It was numb, and I kept shaking it. Then, I started sweating.

I told my manager, “I’m going home. I’m not going to be able to stay.” My manager said, “You don’t look well,” and he called my husband.

My husband came and got me and took me to the emergency room. My blood pressure was 280 over 190. They said, “You’re not going anywhere, so get comfortable.” I ended up having to be admitted. They ran some more tests, and I stayed there for a week.

They did a lung biopsy, and then a right heart catheter. They did a crash blood pressure drop on me, and they would give me blood pressure medication three times a day. I felt like somebody was coming in and beating me.

I said, “Why do I feel this way?” They said, “We have to get your blood pressure down.” I had no idea, because my pressure was always high.

Once I got all the test results back, I met with my pulmonologist. He told me I had pulmonary arterial hypertension, that basically, my heart and lungs are not friends right now. I had no idea that my life was going to change.

I was devastated. I wanted to go back to work. And I couldn’t. I couldn’t even go up the stairs in my house. I either stayed upstairs or downstairs. To walk from my kitchen to my bathroom, I was gassed out. If I got up too fast, I fell out. If I dropped my head too long to tie my shoe, I passed out.

I couldn’t understand it. I got mad. I questioned myself. I questioned God, and I know that wasn’t the right thing to do, but I didn’t know what else to do. I wanted answers. I prayed for understanding.

Then I just started learning. I got on the websites and started learning about PH. There were things I had to do differently. I was confined to my room for six weeks because I couldn’t go up and down the stairs. I had to sit on the stairs and go up backwards. I was a very independent person. Then to have to rely on someone to cook me breakfast, to get me water, I thought, ‘This is unreal.’

I had to reform my thinking. My doctor told me I had to lose weight. And I thought, ‘Well, how is that going to happen if I can’t exercise?’

So I had to learn how to cut back on carbs, sodium, and to fall in love with water. I was 325 pounds, and now I’m 260, and I feel much better than I did a year ago. My doctor actually told me last week, “Compared to how you were last year, you would have never known.”

It just happened. I have had asthma since I was a little girl, but I only had one flare up as an adult. PAH hides behind asthma, and then it explodes all of the sudden. People with asthma have to be very careful and have things checked out extensively. There were no gradual signs.

Every time I would go to the doctor, my blood pressure would be high, and they would ask, “Do you have high blood pressure in your family?” And I would say, “Yes.” And they would ask, “Well, how do you feel?” And I would say, “I feel fine.”

But passing out and having seizures, never had I experienced that before. I was going through my own thing.

I didn’t really want to talk to people, because I couldn’t talk. I was gassing out. The longer I talked, the worse it got. You could hear I was struggling to talk. Last year, I was in a wheelchair.

I was scared, because you read things on the internet about your life expectancy. Then you read about all the new medications that have been FDA-approved. You’re hoping and praying that one of those will work for you.

This year is like a complete turnaround. I have a lot to be thankful for. I’m able to walk distances at my own pace. I still use a wheelchair in big places. I don’t do the mall. I get anxiety around a lot of people, because I feel like, if something happens, I’m not going to be able to get out. I try not to go to a lot of places by myself, and if I go somewhere by myself, I let someone know where I am. I have a Life Alert.

When I walk, I do so at my own pace. If I get tired, I stop. I do all right. [Recently] I went to the doctor by myself. I walked in on my own. But I took my time.

I can’t work anymore—that was the biggest thing. My husband’s work is seasonal. Financially it’s very hard. So, I communicate a lot with other PAH patients. I’m on the website with them. I try to get other ideas from people about what they’re doing.

If I could do volunteer work, that would be good. It’s difficult, because I like working with people with disabilities. But I have to be real cautious about my surroundings. If I catch something, I can’t take anything over the counter.

I have two grown children; they’re 20 and 27. My husband has five. I have four grandchildren who live in Washington, D.C. Last year I couldn’t travel because I was sick. Maybe this year, if the weather and my health permit, we’ll take a little road trip to see them. My parents live in Seattle, but I can’t really visit them because of the altitude of a flight.

My youngest son was here for a year, and he was very helpful, but at the same time, he was a teenager, and I didn’t want him to be stuck in the house with me all summer. He ended up going back to California.

I’ve been working ever since I was 12. I don’t know what a hobby is. After I had kids, I went right back into the workplace.

I have a friend, and we’ll get together and have lunch once a month, and it’s nice. I do like to read. I like romance novels. I really got into talking with other people with PAH in my area. I want to look into a chapter here in Charlotte. Maybe I could do something with them.

My husband was and still is a very big part of my healing process. He always makes sure that my needs are taken care of, no matter what, even cooking dinner after working an eight-hour day. I really do appreciate my husband, and I thank God for him.

To the newly diagnosed, I would say, “It’s going to work out. Don’t get upset, because being upset starts a lot of things, like stress.” I still do that, but I really try not to.

Sometimes I think, ‘What if this gets turned off? What if I lose my house?’ I realized when I was doing that, it was making me worse.

I went through a point where I didn’t want to be bothered with anybody. I was mad. I thought, ‘Why can’t I work?’ I thought my life was done.

Now, I just take everything one day at a time. I don’t let anything bother me. I keep it moving. I want to be able to grab, absorb and use everything you give me to the best of my abilities.

I still call myself a newbie. To the newly diagnosed I would say, “Life still goes on. It’s just that you have to take it slow. It’s about you. Nobody else. That’s how it is.”

Posted in Diseases, Featured, Uncategorized | 8 Comments

Easy Infused Water Recipes

Posted on by Charlie

CVC serves up refreshing, no-fuss recipes for water infused with berries, citrus, mint and more! Each recipe makes one two-quart pitcher.

Strawberry Citrus Mint
  • 1 cup strawberries, halved
  • 2-3 fresh mint leaves
  • 1 medium lime, sliced
  • ½ gallon of water
Combine sliced strawberries, limes, and mint leaves into pitcher. Pour water over ingredients. Refrigerate for at least two hours. Serve over ice.
Refreshing Cucumber Lemon
  • ½ medium cucumber, sliced
  • 1 large lemon, sliced
  • ½ gallon of water
Combine sliced cucumber and lemon into pitcher. Pour water over cucumber and lemons. Refrigerate for at least two hours. Garnish with lemon wedge, serve over ice.
Cool Citrus Infusion
  • ½ large orange, sliced
  • ½ large lime, sliced
  • ½ large lemon, sliced
  • ½ gallon of water
Combine sliced citrus into pitcher. Pour water over citrus fruits. Refrigerate for at least two hours. Garnish with a lime wedge and serve over ice.
Very Berry Mint
  • 1 cup blueberries
  • 1 cup strawberries cut in halves
  • 2-3 fresh mint leaves
  • ½ gallon of water
Combine berries and mint leaves into pitcher. Pour water over berries and mint leaves. Refrigerate for at least two hours. Serve over ice, garnished with a sprig of mint.
Posted in Diseases, Featured, Media Center | Leave a comment

Traveling with Chronic Illness: Q&A with Dr. Bradley Connor

Posted on by Charlie

A consultant to the CDC and a gastroenterologist specializing in gastroenterology and tropical medicine, Dr. Bradley A. Connor has been counseling travelers for more than 30 years. Community asked Dr. Connor for his tips on traveling with chronic illness.

Bradley A. Connor, M.D., Medical Director, The New York Center for Travel and Tropical Medicine, Clinical Professor of Medicine, Weill Medical College of Cornell University.

Bradley A. Connor, M.D., Medical Director, The New York Center for Travel and Tropical Medicine, Clinical Professor of Medicine, Weill Medical College of Cornell University.

What do you think is important for those with chronic illness to know about travel?
The ability of people with chronic illness to travel has probably never been better than it is now. The ease of travel has developed to the point where people are going to more and more exotic places because there are physicians and health professionals who specialize in health issues related to travel. Those with chronic disease can feel confident at least knowing what the risks are and taking steps to prevent them.

What are some general tips for those traveling with chronic illness?
• Before you embark on travel—it could be domestic travel, travel to Europe, or a more adventurous type of trip, like a safari in Africa, or travel to Asia—check with your doctor who takes care of your chronic disease and make sure it’s his or her opinion that you’re fit to travel and that your condition is in a stable place.

• If you’re traveling to an exotic destination, avail yourself of travel medicine specialists who know the specific health risks. In some cases, either your own physician or a travel medicine physician might find a physician at your destination to refer you to should your illness become active when you travel.

• Preplanning is absolutely necessary. Make sure that your medications are up to date, and that you have more than enough medication for the trip. If your trip is for two weeks, take four weeks’ worth of medication. We’ve had patients who were stuck in Europe in 2010 when the clouds of volcanic ash from Iceland delayed flights. Be a little bit over-prepared.

• Take your medication, in the original, labeled container, in your carry-on luggage, never in your checked bags.

• If you need to, consult with a travel medicine specialist if you’re going to a place with a destination-specific illness.

Molokai sea cliffs, Hawaii. Photo: Hawaii Tourism Authority (HTA)/Tor Johnson.

Molokai sea cliffs, Hawaii. Photo: Hawaii Tourism Authority (HTA)/Tor Johnson.

• Have an emergency plan in place. What happens if your illness becomes active? What are you going to do? That could be something as simple as an email to your doctor; it could be having a doctor then referred to you at the destination.

• Post-travel, is there anything likely that you’ve picked up on the trip that may impact your illness? What are your doctor’s assessments?

What should travelers with chronic illness know about health insurance?
Check whether you do have health coverage overseas. In many cases you don’t. It’s a good idea to investigate what types of policies you can obtain for your travel in case you need to access medical care.

Some might want to consider medical evacuation services. It’s not for every traveler, but sometimes it’s less expensive to contract with a company like Global Rescue or International SOS in the eventuality that you have to be evacuated back home, or somewhere you can get good medical care. The cost of a policy for a short trip is usually fairly reasonable.

Some medications that are legal here are illegal in other countries. Do these issues come up for travelers with chronic illness?
Those issues do come up. There are certain countries, where certain medications (in some instance, even non-prescription medications) are not permitted into the country. Now, having said that, I don’t know of any instance where a traveler with a prescription medication with a physician’s letter has been denied entrance to the country, or has had their medication confiscated.

But, I think it’s something you at least need to consider. Bring a letter from your physician in case you’re questioned about your medication.

In some countries, if you have a physician there who is prepared to give you the medication or prescribe it there, that’s another way around it. But that takes a lot of effort.

The other issue, which is very important, is that the world is plagued with counterfeit medications. Beware of counterfeit medications sold in other countries. If you see a drug that sells for $10,000 here selling somewhere else for $400, be careful. People should take more than enough medication and not plan on buying medication overseas.

What are some of the precautions those who are planning to go on cruises should take?
One thing they can do is to check out the medical backup at the various cruise lines. Everyone is interested in keeping passengers healthy. Some do a better job than others. There are a few cruise lines that have put a lot of effort into having excellent physicians onboard, and having excellent medical directors.

With the norovirus outbreaks that you see on cruise ships, it’s not that there’s a lapse of hygiene with the cruise lines, it’s just that you have a lot of people together, and people are coming onboard with illness. Take the usual, frequent precautions for hand hygiene, like washing with soap and hot water and using hand sanitizers, and make sure the medical back up is there. Do the research.

What are some considerations when traveling with oxygen?
• If you plan on flying, ask your doctor whether you can tolerate the reduced air pressure on a plane and if you’ll need an additional oxygen supply during the flight.

• Check with your airline well in advance of travel about regulations and methods for permitting supplemental oxygen, including the need to carry a prescription for your portable oxygen compressor, whether it’s an approved device, and whether the airline charges for providing supplemental oxygen.

• If you’re on supplemental oxygen, make sure you have an adequate supply and that you have extra batteries for your POC in your carry-on luggage.

• If you’re going to a destination with high altitude, it’s important to let your physician know. Discuss with your doctor what effects altitude might have on your illness and on the delivery of oxygen.

Le Train Bleu, restaurant Gare de Lyon, Paris. © Paris Tourist Office. Photo by Amelie Dupont.

Le Train Bleu, restaurant Gare de Lyon, Paris. © Paris Tourist Office. Photo by Amelie Dupont.

What would you say to those with chronic illness who want to travel, but are afraid?
With proper planning, some of the perceived restrictions of traveling don’t have to be. Use your doctor as a partner. Use specialists in travel medicine if need be. Have an escape plan in place if you get sick. Don’t leave anything to chance.

If you’re new to traveling with chronic illness, take an easy trip first. See that you can do it. Then, maybe for your next trip, you can be a little more adventurous.

Done the right way, with a lot of preplanning, you don’t have to fear travel. If you’re suffering with a chronic illness, travel can be very enlightening and it can be uplifting psychologically.

Posted in Diseases, Featured, Media Center | Leave a comment

Gluten-Free World Tour Part II

Posted on by Charlie

Let these delicious internationally-inspired gluten-free recipes from top chefs around the world whet your appetite for new dishes and destinations. Eva Leonard gets the details on restaurants serving up gluten-free goodness.

Chef Chris Clime

Chef Chris Clime. Photo by Scott Suchman.

When Chef Chris Clime’s wife was diagnosed with the autoimmune disease lupus, she embarked on a strict gluten-free diet on the advice of her doctors. It was then that Clime began to notice a growing demand for gluten-free dishes at PassionFish, the Reston, Virginia, restaurant where he is executive chef.

“Maybe it was a coincidence, but more likely, my awareness was heightened by our personal situation,” says Clime. “The good news is that my wife’s condition is not life-threatening, and going gluten-free has been really good for her, as well as the rest of the family.”


Many dishes on PassionFish’s regular menu already meet gluten-free criteria, and Clime is happy to accommodate special requests. “It’s always been fun for me to create with new or different ingredients, so this trend is a challenge I don’t mind at all.

“Some of our most popular dishes are Asian-inspired, and since Asian cuisine is rice-based, wheat flour isn’t much of an issue. The red Thai curry lobster clay pot and the crispy Vietnamese crab and shrimp spring rolls are naturally gluten-free. So is the whole crispy flounder, which is prepared with cornstarch, rather than flour.”

Clime—a big fan of Latin flavors—notes that Latin cuisine is not very wheat flour-heavy, often using corn or cassava flour instead. He coats PassionFish’s New Orleans fried oysters and shrimp in corn flour and has adapted other menu items to eliminate gluten, including jumbo lump crab cakes, and blue crab and corn chowder, which he now makes with rice flour.

At home, Clime experiments with a range of flour alternatives—the Climes now eat pasta made with corn flour. Clime’s wife’s heritage is Indian, and the family also enjoys various Indian dishes with roti, a flatbread made with lentil flour.

Clime’s favorite flour alternative for coating chicken and veal for scaloppini is a chickpea/almond/amaranth flour combo available at Trader Joe’s. “The crust gets crispy like it’s been deep-fried, although it’s just sautéed. That’s a pretty cool discovery that I never would have made if I hadn’t been forced to expand my flour horizons.”

Gluten-Free Jumbo Lump Crab Cakes
and Sweet Corn Chow-Chow
Chris Clime, Executive Chef, PassionFish, Reston, Virginia

Ingredients Yield: Four crab cakes

Gluten-Free Jumbo Lump Crab Cakes

Gluten-Free Jumbo Lump Crab Cakes. Photo courtesy of PassionFish.
  • 1 pound Maryland jumbo lump crabmeat
  • 3 dashes Tabasco sauce
  • 1 each lemon, juiced
  • 1 tablespoon Old Bay seasoning
  • 1 tablespoon chives, chopped
  • 2 teaspoons kosher salt
  • ½ cup mayonnaise
  • 1 each egg [large]
  • 1 tablespoon rice flour
  • 1 tablespoon grape seed oil
  • - To taste, drawn butter
  • - To garnish, chives, chopped


  • Carefully pick the crabmeat [see suggested procedure below] and refrigerate until needed.
  • Next, in a mixing bowl, combine Tabasco, lemon juice, Old Bay, chives, salt, mayonnaise, and egg. Mix until all ingredients are well combined.
  • Gently add about two-thirds of this mixture to the chilled crabmeat, carefully folding it in so you don’t break up the lumps.
  • Check the seasoning; you want to add just enough wet mixture to the crab to season it well, without adding so much that you have to add extra rice flour.
  • Add just a bit more of the wet mixture if needed, and then gently add in the rice flour to bind the mixture together.
  • Cover the bowl with plastic wrap and refrigerate for 30 minutes.
  • Preheat the oven to 350°F.
  • Divide the mixture into four portions and form crab cakes. [Note: You can make mini crab cakes if preferred.]
  • Add about one tablespoon of grape seed oil to a non-stick pan and sauté the cakes until golden brown on one side.
  • Flip the cakes and sauté on the other side until golden brown.
  • Transfer the cakes to a baking sheet.
  • Top each with a little drawn butter and finish in the preheated oven for 5-7 minutes. Do not overcook; finishing them in the oven is just to help the cakes bind together and plump the meat.


  • Serve atop sweet corn chow-chow [see recipe below] and finish with tartar sauce [if desired] and freshly chopped chives.

Ingredients for the sweet corn chow-chow

  • 3 each yellow or white corn ears, whole
  • 3 tablespoons extra virgin olive oil
  • ¼ cup apple cider vinegar
  • ¼ cup red bell pepper, diced
  • ¼ cup red onion, diced
  • 1 teaspoon cilantro, chiffonade
  • - To taste kosher salt
  • - To taste cracked black pepper


  • Roast the corn in the husks at 450°F for 20 minutes.
  • Then shuck and slice off the ear.
  • Combine all ingredients in a bowl, and season to taste.

Method to pick crabmeat

  • Fill two bowls with ice and place a smaller bowl on top of each ice bath.
  • Place the crabmeat into one of these smaller bowls to keep it chilled while you clean it.
  • Fill this bowl with cold water.
  • Grab small lumps of crabmeat and gently pick through the tops to remove any cartilage, being careful not to mash the meat or break up the lumps.
  • To pick the shell out, dip your fingers in the water so the shell will stick to your fingers as you clean the meat.
  • To remove the shell, dip your fingers into the water so it will go into the bowl.
  • Place the cleaned meat into the other bowl.


Fairmont Hotels & Resorts’ more than 60 properties around the globe include the Fairmont, Raffles, and Swissôtel brands. The hotel group’s Lifestyle Cuisine Plus menu caters to guests with specific diet-dependent conditions such as diabetes, heart disease and gluten-free, as well as dietary preferences including, macrobiotic, raw and vegan.

Fairmont chefs have been trained to prepare a wide range of special dietary and allergy-specific meals and use recipe analysis software to help customize entrees and menus to meet guests’ caloric and nutritional requirements. Using nutrient-rich ingredients, clean cooking methods and local food products, chefs create dishes that are wholesome, balanced and full of taste. Guests with food allergies and sensitivities are invited to speak with the chef to plan their food options during their stay.

Gluten-Free Golden Apple Salad
Raffles Praslin, Praslin Island, Seychelles

Ingredients Yield: One serving

  • 120 grams golden apple
  • 10 grams sugar
  • 2 small chilli shots
  • 1 tablespoon olive oil
  • 1 tablespoon white wine vinegar
  • 1 tablespoon spring onion
  • 2 leaves of mint
  • 20 grams red onion


  • Wash, peel with peeler and grate the golden apples with grater.
  • Squeeze them in the strainer until dry and remove the juice.
  • Pre-heat sauté pan on the stove, add olive oil and let the pan warm.
  • Add chopped onions and sauté until they get the aroma for 1 minute and add the golden apples.
  • Sauté for 3 minutes, remove from fire and transfer to a bowl.
  • Add brunoise chili, squeeze lemon juice and white vinegar.
  • Mix all ingredients and seasoning with salt and pepper.
  • Ready to serve.
Gluten-Free Braised Buffalo Short Ribs With Heirloom Tomato & Apricot Chutney
Fairmont Banff Springs Hotel, Alberta, Canada
Gluten-Free Braised Buffalo Short Ribs with Heirloom Tomato & Apricot Chutney

Gluten-Free Braised Buffalo Short Ribs with Heirloom Tomato & Apricot Chutney. Photo courtesy of Fairmont Hotels & Resorts.

Ingredients Yield: 10 servings

  • 10 (6-inch) buffalo short ribs (about 9 pounds total)
  • 8 cups red wine
  • 1 bunch fresh thyme
  • 1 bunch fresh flat-leaf parsley
  • 2 fresh bay leaves or 1 dried
  • 1 tablespoon plus 2 teaspoons freshly ground black pepper
  • 1 head garlic, peeled and coarsely chopped
  • 2 tablespoons kosher salt
  • 3 cups onions, peeled and cut into ½-inch pieces
  • 2 ½ cups carrots, peeled and cut into ½-inch pieces
  • 2 ½ cups celery, leaves removed and cut into ½-inch pieces
  • 6 cups beef jus
  • 2 cups peeled pearl onions
  • 2 cups sherry vinegar
  • ½ cup raw cane sugar
  • 3 cinnamon sticks
  • - juice and zest from one orange

Method for short ribs

  • Add wine, thyme, parsley, bay leaves, 1 tablespoon pepper, and garlic.
  • Place in large bowl and marinate in refrigerate for 24 hours.
  • Remove ribs from marinade and drain, reserving liquid.
  • Pat ribs dry and sprinkle with kosher salt and remaining 2 teaspoons pepper.
  • Pre-heat oven to 300°F.
  • Heat a large cast iron skillet, over moderately high heat, heat ¼ cup oil just until smoking.
  • Transfer ribs, bone side up in a roasting pan and set aside.
  • Add onions, carrots, celery to skillet, sauté until lightly caramelized.
  • Add over ribs, pour stock and cover with foil, braise in oven for 3-4 hours or until meat pulls away from bone.
  • Remove from oven and allow to cool.
  • Drain off liquid into a heavy- bottom sauce pot and skim fat.
  • Reduce to coat back with spoon.

Method for pearl onions

  • In a heavy bottom sauce pot add vinegar, sugar, cinnamon stick and fresh thyme.
  • Bring to a boil.
  • Add orange juice and zest, add onions and simmer for 8 minutes, take off stove and allow to cool.
  • Place in a bowl.

Ingredients for tomato and apricot chutney

  • 2 cups diced tomato
  • 2 garlic cloves, finely chopped
  • 2 teaspoons finely-chopped peeled fresh ginger
  • 1 tablespoon vegetable oil
  • 1 teaspoon mustard seeds
  • ¾ cup dried apricots (6 ounces), finely chopped
  • ⅓ cup dried currants (1 ½ ounces)
  • ¾ cup water
  • ⅓ cup red-wine vinegar
  • ¼ cup sugar
  • ¾ teaspoon salt


  • Cook garlic and ginger in oil in a 1 to 1 ½-quart heavy saucepan over moderate heat, stirring, until golden, 1 to 2 minutes.
  • Add mustard seeds and cook, stirring, until fragrant, about 1 minute.
  • Add remaining ingredients and simmer, partially covered, stirring occasionally, until almost all liquid is absorbed, about 20 minutes (chutney should be moist).
  • Cool to room temperature.

Gluten-Free Golden Apple Salad. Photo courtesy of Fairmont Hotels & Resorts.
Gluten-Free Sirloin Steak with Mango Salsa
Restaurant, The Fairmont Kea Lani, Maui

Ingredients Yield: One serving

  • ½ small mango
  • ½ organic lime
  • 1 ½ spring onions
  • 5 mint leaves
  • - salt
  • 1 sirloin steak (about 180 grams)
  • 1 tablespoon mixed pepper
  • 1 tablespoon olive oil


  • Season sirloin with salt and crushed pepper and keep for 5 minutes.
  • Light the grill or preheat a grill pan.
  • Grill sirloin steak over moderate heat, turning and making mark on top of the beef until it is cooked as per taste.
  • Wash, peel with peeler and boil the potatoes until tender.
  • Throw the water and transfer the potatoes into a potato masher.
  • Reheat the pan and add cooking cream; bring to boil and add the potatoes.
  • Stir with balloon whisk until smooth and creamy.
  • Season with salt and pepper and transfer to piping bag.
  • Keep for 3 minutes and present as a line on the plate.
  • Prepare boiling water in the pot on the stove.
  • Wash and peel all vegetable ingredients and cut into jardiniere.
  • Put all the vegetables for blanch for 3 minutes, strain and put into a bowl with ice water.
  • Preheat sauté pan, add olive oil and sauté vegetables until they get aroma, add seasoning: salt and pepper.
Gluten-Free Sirloin Steak with Mango Salsa

Gluten-Free Sirloin Steak with Mango Salsa. Photo courtesy of Fairmont Hotels & Resorts.

Method for mango salsa

  • Wash and peel the mangos with a vegetable knife.
  • Peel the onions and wash properly.
  • Prepare the cutting board and cut mangos and onions into brunoise.
  • Thinly slice the coriander.
  • Put the onions and mango into a bowl and add the sweet chili and coriander.
  • Ready to serve and garnish with cherry tomato.
Gluten-Free Chocolate Flan with Berries
Fairmont The Queen Elizabeth, Montréal, Québec, Canada
Gluten-Free Chocolate Flan with Berries

Gluten-Free Chocolate Flan with Berries. Photo courtesy of Fairmont Hotels & Resorts.

Ingredients for the chocolate ganache Yield: 10 servings

  • 400 grams non-dairy dark chocolate
  • 3.7 ounces maple syrup
  • 350 grams silken soft tofu
  • 1 tablespoon vanilla extract
  • 1 pinch salt
  • Ingredients for the unbaked crust
  • 650 grams pecans (chopped and roasted)
  • 350 grams dried apricots and figs
  • 20 grams sugar
  • 5 centiliters chocolate sauce
  • 20 blueberries
  • 30 raspberries
  • 10 mint leaves

Method for the chocolate ganache

  • Pour the ingredients in blender and stir in the melted chocolate.

Method for the unbaked crust

  • Roast and chop the pecans.
  • Cook and purée the dried fruits.
  • Mix together and press into the bottom of cake ring.
  • Pour ganache over it and let it cool down.
  • Serve with a little bit of chocolate sauce and berries.
  • Decorate with mint leaves.

Click here to read part 1!

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Gluten-Free World Tour Part I

Posted on by Charlie

Let these delicious internationally-inspired gluten-free recipes from top chefs around the world whet your appetite for new dishes and destinations. Eva Leonard gets the details on restaurants serving up gluten-free goodness.

Chef Tori Kramer

Chef Tori Kramer. Photo courtesy of Park Hyatt Washington.

Tori Kramer, pastry chef for the Park Hyatt Washington, D.C.’s award-winning Blue Duck Tavern, describes the evolution of her gluten-free awakening.

“I worked in a pie shop, and I was around a lot of flour. When I was in the shop, I got sick, and had to wear a mask. A friend suggested I give up gluten, but there was no way I was going to leave the kitchen—baking is my passion. Now, I’m in a larger kitchen, where the ventilation is great, and we make gluten-free and vegan items.”

Among those delectables: a carrot apple muffin topped with pecan streusel that has no refined sugar, but derives its natural sweetness from apples, carrots and applesauce, a multi-textured, chewy cherry-chocolate-almond granola bar, and a rich, moist orange-glazed orange scone. Kramer’s gluten-free creations make the palate feel treated, rather than deprived.

“Once I found out I had celiac disease, I went to the store, and I was completely distraught. That’s when I made it my mission to create delicious, gluten-free food,” says Kramer.

“The first gluten-free thing I made was cookies, and that was really exciting. I made my grandmother’s bread recipe and had to play around with it. I spent an entire weekend baking nothing but bread.”

Kramer’s recipes have been designed with maximizing spurts of energy in mind: “You can make it in big batches and keep it in the freezer. It’s simple and still delicious. When I did make food for myself, I had so little energy.

“For me, the most important thing has been to remove the stigma of gluten-free food. I want to be able to make one meal that everybody can enjoy. We’re making huge progress in the world of gluten-free. We’re not such a tiny minority anymore.”

An enthusiastic response to Blue Duck Tavern’s gluten-free options, Kramer notes, has prompted the restaurant to expand its menu to include items like gluten-free waffles and pancakes and fueled her desire to cater to other dietary needs.

“Now that we’ve been receiving great feedback from the gluten-free menu items, it’s so important to open this up for those with other dietary restrictions.”

The biggest challenge Kramer says she has found is in trying different ingredients. “I’m still learning every single day. You have to take into account texture, feel, and taste. You have to keep trying and learn to make things in big batches when you find the time and energy.

“The most exciting thing for me is when I create a gluten-free menu—to be able to bring my knowledge here and share that with everyone else.“

Gluten-Free Carrot Apple Muffins Topped with Pecan Streusel
Tori Kramer, Pastry Chef, Blue Duck Tavern, Park Hyatt, Washington, D.C.

Ingredients Yield: 15 muffins

  • 2 cups gluten-free flour (see ingredients for gluten-free flour below)
  • 1 tablespoon baking powder
  • ½ teaspoon baking soda
  • 1 teaspoon salt
  • ½ teaspoon ground ginger
  • ½ teaspoon ground cinnamon
  • ¼ teaspoon ground cloves
  • 1 cup unsweetened applesauce
  • ½ cup granulated sugar
  • ½ cup light brown sugar
  • 1 teaspoon vanilla extract
  • 2 large eggs
  • 1 cup finely shredded carrots

Ingredients for gluten-free flour

  • 4 cups white rice flour
  • 4 cups brown rice flour
  • 4 cups corn starch
  • 2 cups tapioca flour
  • 2 cups potato starch
  • 2 ½ tablespoon Xanthan gum

Method for gluten-free flour

  • Mix all together well

Method for muffins

  • Combine first 7 ingredients and mix well.
  • Cream applesauce and sugars together in mixing bowl with paddle.
  • Add vanilla and eggs one at a time until blended.
  • Add dry ingredients on low just until blended.
  • Fold in carrots. Let sit for 40 minutes—overnight (in fridge).
  • Scoop into greased/lined muffin tins, sprinkle top with streusel and bake at 350°F for about 15-20 minutes until toothpick inserted in center comes out clean.

Ingredients for pecan streusel

  • 2 tablespoons gluten-free flour
  • ¼ cup light brown sugar
  • ¼ cup chopped pecans
  • ½ teaspoon cinnamon
  • 1 tablespoon cold butter (cut into small cubes)
  • Method for pecan streusel
  • Mix all together until crumbly.

Gluten-free muffin and scone

Photo courtesy of Blue Duck Tavern.
Gluten-Free Orange Scones with Orange Glaze
Tori Kramer, Pastry Chef, Blue Duck Tavern, Park Hyatt, Washington, D.C.

Ingredients Yield: 20 large scones (4 to 5 inches wide each) or cut ingredient amounts in half for 10 scones (2 to 2.5 inches wide each)

  • 6 pounds gluten-free flour
  • 1 pound sugar
  • 3 ounces baking powder
  • 1 ounce salt
  • 2 pounds butter
  • 1 ½ quarts cream
  • 10 eggs
  • - zest of three oranges

Method for the scones

  • Use the cutting in method to obtain pea-sized pebbles of butter, add all liquid ingredients and mix just until combined. Do not over mix.
  • Chill in a 1 ½–2 inch thick packet.
  • Cut out triangles and egg wash with egg or cream and bake at 325°F until just baked in the center, about 15 minutes.

Ingredients for the orange glaze

  • 1 cup confectioners’ sugar
  • 1 teaspoon vanilla extract
  • 1 tablespoon baking powder

Method for the orange glaze

  • Whisk all together until smooth and drizzle over scones.


Chef David Guas

Chef David Guas. Photo by Jonathan Times.

New Orleans native and award-winning chef and owner of Arlington, Virginia’s Bayou Bakery, Coffee Bar & Eatery, David Guas was recently tapped to host the new competitive outdoor cooking challenge show, American Grilled, debuting this summer on the Travel Channel. Oprah Winfrey’s O Magazine named Guas one of the “Ten Best Pastry Chefs in the Country,” and his cookbook, DamGoodSweet—Desserts to Satisfy Your Sweet Tooth New Orleans Style was one of Food & Wine magazine‘s “Best New Dessert Cookbooks” in 2009. Bayou Bakery, Coffee Bar & Eatery began offering gluten-free items, including the chewy honey almond bars below, two years ago.

Gluten-Free Chewy Honey Almond Bars
David Guas, Chef/Owner, Bayou Bakery, Coffee Bar & Eatery, Arlington, Virginia

Gluten-free Chewy Honey Almond Bars

Gluten-free Chewy Honey Almond Bars. Photo courtesy of Bayou Bakery, Coffee Bar & Eatery.

Ingredients Yield: 12 bars

  • 3 ounces unsalted butter
  • 5 ounces light brown sugar
  • 4 ounces honey
  • 2 ounces peanut butter, smooth
  • 1 tablespoon vanilla extract
  • 2 ¼ cups oats, old-fashioned
  • 1 ½ cups Rice Krispies cereal
  • 1 cup unsalted almonds
  • 1 cup raisins
  • 1 teaspoon salt, kosher


  • Add the first five ingredients to a small two-quart stockpot.
  • Place the remaining dry ingredients in a stainless steel mixing bowl and reserve.
  • In the stockpot with a candy thermometer, stir all ingredients together with a rubber spatula on medium-high heat.
  • Simmer and cook until the mixture reaches 220°F.
  • Pour mixture slowly over the top of reserved dry mix and stir with rubber spatula until all dry ingredients are coated.
  • In a 13×9-inch baking pan, place parchment paper down and spray lightly with Pam before pressing the bar mixture down into the pan by hand. Then place the filled pan in the refrigerator for about an hour.


  • Remove after refrigerating and cut the bars length-wise in half.
  • Then cut bars just over 2 inches wide for a total of 12 bars.


Stay tuned for Part 2 of our Gluten-Free World Tour, highlighting more gluten-free recipes from top chefs!

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