The drug, from biotech company Sarepta Therapeutics, will need to prove in an ongoing clinical trial that it improves physical function and mobility in patients with Duchenne muscular dystrophy in ...

COLORADO SPRINGS, Colo. (KRDO) -- A groundbreaking gene therapy was recently approved by the U.S. Food and Drug Administration to treat the most common form of muscular dystrophy in children, Duchenne ...

(CNN) — The US Food and Drug Administration has approved the first gene therapy to treat a rare and devastating muscle disease but limited the approval to kids ages 4 and 5 based on available evidence ...