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