Category: Muscular Dystrophy

FDA Approves Sarepta’s Gene Therapy for Rare Muscular Dystrophy in Some Kids

The U.S. health regulator has granted accelerated approval to Sarepta Therapeutics’ first-of-its-kind gene therapy for Duchenne muscular dystrophy (DMD), an inherited progressive muscle-wasting disorder that almost always affects young boys. Sarepta said on Thursday the Food and Drug Administration had approved the treatment for children aged between 4 and 5 years who can walk. It…


Woman With Rare Form of Muscular Dystrophy Defies Doctor by Hitting the Gym

Getting out of bed in the morning can be difficult for some, a deep sleep suddenly disturbed by a blaring alarm coming from a smart phone in a demand for you to start your day. For Lily Brasch, getting out of bed in the morning presents an entirely different kind of challenge. Brasch, who turns…