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

Fda Food And Drug Administration Health And Human Services

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.

Read the full story on AP News right here.

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

Categories: Regional and US News