The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

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

The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb-girdle ...

Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the FDA.

Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...

Separately, the FDA on Friday informally requested that Sarepta voluntarily pause shipments of another gene therapy, Elevidys, that is approved to treat a different type of muscular dystrophy and was ...

Sarepta Therapeutics said it will resume shipments of its gene therapy Elevidys® (delandistrogene moxeparvovec-rokl) “imminently” to ambulant patients with Duchenne muscular dystrophy (DMD), ending a ...

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

Sarepta Therapeutics has paused U.S. shipments of its gene therapy Elevidys after two teenagers with Duchenne muscular ...