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

The Associated Press

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 a long list of concerns from some FDA scientists about the company’s research. But patients, physicians and and parents urged approval at a public meeting 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.

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6/22/2023 4:40:26 PM (GMT -5:00)