Dystonia causes involuntary muscle contractions and can result in abnormal posture. There are a number of different types of dystonia, and there is no known cure. Parkinson's disease (PD) is…
A recent report from the British Heart Foundation (BHF) stated many more people in the UK are at risk for early heart attacks than previously thought. The culprit? High cholesterol. And not the…
Okay, people in the CF Community, listen up! A new targeted treatment for Cystic Fibrosis (CF) may be on the horizon sooner vs. later, and I’m pretty freaking excited about it! This…
Although it is often called the "Most Wonderful Time of the Year", people can get pretty up-in-arms about holiday lingo and traditions. If you don't believe us, just ask Starbucks...…
There are actually three types of the disease called Tyrosinemia, but in this post, we're only going to focus on Type I. Tyrosinemia, type 1 (TYR 1) is a genetic disorder…
In the past, 14-year-old Louise Stewart-Scott, was used to flying through the air and across the mat during her gymnastic routines. But, at the age of eight, she was diagnosed…
We know that the cost of medical treatment, even with great insurance, is high. Transportation, parking, inability to work full-time because you are taking care of a very ill family…
Whether you think so or not, you have the strength to live well with Myasthenia Gravis (MG). But for your times of doubt and frustration, it can be helpful to have…
Shelby Klug, a 17-year-old from LA, was diagnosed with cystic fibrosis (CF) at age 2. Cystic fibrosis is a genetic disorder that causes damage to the lungs and digestive system.…
If you have cystic fibrosis, you might know that it is linked to low PH levels. The current PH sensors aren't sensitive enough to capture molecules at different stages called…
"As far back as I can remember, i was told pain was normal, or in my head, and to keep going" Welcome to the life of Dawn Michelle Shepley...and,…
Sam Bradley hit the dystonia jackpot, but that's not a good thing. Bradley won the NCAA record for the quickest (wrestling) pin in college, and is a black belt in karate,…
Brothers Michael, 6, and Dylan Cavalier, 4, aren’t exactly like other boys. They don’t make mud pies; they don’t jump in piles of autumn leaves; and they certainly don’t dig…
Back to our interview with Mary! Miss the first part? Click here. Mary:- "I think education is power and the more you know about your disease process the more prepared…
Not long ago, Vertex Pharmaceuticals scored a big one for their investors—the European Commission has approved the use of ORKAMBI® (lumacaftor/ivacaftor), for the treatment of cystic fibrosis. This breakthrough treatment will be used…
Researchers in rare disease find it very challenging to find specimens. The Newborn Screening Translational Research Network (NBSTRN), has developed an easy way for researchers to access specimens through the…
Hey, want to know how to really ruin the day for someone living with excessive daytime sleepiness (EDS)? Just tell them “Oh, I get tired a lot too.” Apart from…
News services recently picked up a fascinating story that I think everyone should read! Little 8-year-old Kayleigh Petersen, from East Brunswick, New Jersey, stunned a packed crowd of loyal hockey…
So here’s a nice antidote to all those stories about greedy pharma execs soaking patients for ultra-expensive treatments. There’s a new approach to contrpay-acting between insurance companies and pharmaceutical companies…
This month, I came across the most refreshing article written by Caroline Newman and featured in UVA Today's Art & Culture section about one of the longest living cystic fibrosis survivors.…
Mary is a former registered nurse, a mother to four daughters, a wife, and a grandmother to one grandson as well as several “grandpets”. She’s an avid gardener; growing cherry…
The Oley Foundation is a lifeline of support for those dependent on home IV nutrition (HPN) or tube feeding (HEN). There are many causes for the inability to adequately nourish…
If you have narcolepsy, excessive daytime sleepiness, and/or cataplexy, you know how hard it can be to function in "real-world" time. And while treatments have improved, there is still no…
Over the past few years, Casey Higgins, a 27-year-old Ohio woman, has required supplementary oxygen 24/7 to help her breathe. Diagnosed with the genetic disease cystic fibrosis at the age of…
Robert began his mission to raise awareness of Cystic Fibrosis after getting to know someone who had lived with it for forty one years. As he puts it; “She inspired…
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