US 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 was initially seeking approval for all DMD patients who can walk.

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