A clinical trial is up for those suffering from Duchenne Muscular Dystrophy. An oral tablet called Idebenone will be compared to a placebo, to see if it is safe and…
2017 Muscular Dystrophy Association Scientific Conference was a beacon of light! Researchers from academic medical centers, pharmaceutical companies, children’s’ medical centers, biotech, engineering, and veterinary medicine came together to share…
I recently covered an author who had some solid advice for drug makers in regards to the rare disease pricing problem. The following drug maker may or may not have…
One tie, two ties, red tie, blue tie... The list goes on and on for Doug Robins, a young Australian man who’s raising awareness for Duchenne muscular dystrophy (DMD) by…
Welcome back to Editor's Choice Patient Worthians! This week, a PW Contributor told a love story about how her husband's bipolar disorder helps her manage her rare disease. We also have an…
What at first glance sounded like nothing but good news, the FDA's approval of an Duchenne Muscular Dystrophy drug, has complicated undertones. It was announced last week that Emflaza (deflazacort) was approved…
If you’re like most people (especially me), you’ve never heard of nicotinamide riboside (NR) before. But that may change very soon, especially for the Duchenne muscular dystrophy (DMD) community. What…
Duchenne Muscular Dystrophy (DMD) is a degenerative disease that causes muscle weakness in the hips, thighs, shoulders and then later, in the heart and respiratory systems. This Australia family is…
So many workouts, so many excuses. Running? Cramps in your side. Swimming? Hate getting wet. Pilates? Expensive. Spin class? A headache. Walking? Um... Finally, an exercise truly devoid of downsides.…
Years ago, there was a television show called Tales from the Crypt. From what I remember, a creepy-looking hand would open a creaky-sounding door and the viewer would "enter" the…
A few months ago, I went on a rant about the FDA's failure to approve a drug that would benefit children with Duchenne muscular dystrophy, or DMD, a muscle-wasting disease.…
Talking to the parents or family of a child with a serious illness can be nerve-racking and uncomfortable. Sometimes avoiding the family can seem easier than facing a difficult situation.…
Any of you ever think about running a marathon? I know I have. I've thought about it. Have I run a marathon? NOPE! Sure haven't. That sh*t looks hard. Look,…
On the surface, it seems self-evident that social collaboration can have a positive impact--especially when it comes to Duchenne muscular dystrophy (DMD). Working together with all invested participants to influence…
If you, or someone you love, has Duchenne muscular dystrophy (DMD), then each new possible drug that's brought before the FDA brings with it a sense of hope. Race to…
When you were a kid and planning your future, what events did you imagine? Your first car? Your prom? Your graduation? Your wedding? For this boy with Duchenne muscular dystrophy, he…
If a treatment that would cure me or my child had to use dogs for testing, what would I do? That’s the question I had to ask when I saw…
Netflix recently released an original dark comedy that features a young boy from the UK with Duchenne Muscular Dystrophy whose caregiver (played by Paul Rudd) is new at his job and…
It's no easy battle, but for Tayjus Surampudi and many others, muscular dystrophy is a battle worth getting up and fighting against. Tayjus is a student at Harvard. And if…
For 21-year-old Tanner Pyeatt, the future is uncertain. Diagnosed with Duchenne muscular dystrophy at the age of four, he gradually lost the use of his legs and has been wheelchair-bound since age nine.…
If you have a rare disease with no cure, it’s important to live your future today. Bradon Coy, 10, took that advice by travelling all the way from the United…
It’s Friday afternoon and I’ve been racing around at work all day pecking wildly at my PC to get my stuff done, and just as I was about ready to…
Anyone with rare and neurological disease can relate: there aren't a ton of treatment options and there are almost never any cures for our conditions. We often times just have to treat…
Last year, the FDA approved a record number of new drugs to treat rare diseases, according to the National Organization for Rare Disorders (NORD). In all, 21 "orphan" drugs were…
Q: What do Fergie, Josh Dumal and Scott Niedermayer have in common? A: They are celebrity spokespeople for CureDuchenne a nonprofit organization that raises awareness and funds research to find a…
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