The Food and Drug Administration placed a clinical hold on Sarepta Therapeutics' investigational gene-therapy clinical trials for limb-girdle muscular dystrophy, a group of genetic disorders that ...

Sarepta Therapeutics faces challenges as the FDA halts gene therapy trials for LGMD and revokes AAVrh74 platform designation.

Also on Friday, the Food and Drug Administration informally requested that Sarepta voluntarily pause shipments of another gene therapy, Elevidys, which is approved to treat a different type of ...