Sarepta Therapeutics has rejected a call by the Food and Drug Administration to stop shipments of its muscular dystrophy drug ...

FDA places clinical hold on Sarepta's LGMD gene therapy trials after three deaths and revokes platform tech designation due to safety concerns.

Although Sarepta initially appeared to have avoided the worst possible outcome for Elevidys—a market withdrawal—thanks to the gene therapy’s new black box warning, the FDA is signaling stronger ...