First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids

By MATTHEW PERRONE
AP Health Writer

WASHINGTON (AP) — The U.S. Food and Drug Administration has approved the first gene therapy to treat a deadly form of muscular dystrophy. The treatment from Sarepta Therapeutics was approved Thursday for children ages 4 and 5 with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes early death. The approval came despite concerns from some FDA scientists about flaws in the company’s research, including its lead study which failed to show an improvement in measures like standing and climbing. But patients, physicians and and parents urged approval at a public meeting on the therapy in April. The drug received accelerated approval, meaning the FDA has the option to revoke its use if further studies don’t confirm it works.