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

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

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

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

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

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 ...

U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a ...

Sarepta Therapeutics’ share price has fallen again following the death of a third patient dosed with its gene therapy treatment.

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 ...

A third fatality has been recorded among patients receiving treatment with a Sarepta gene therapy, adding to the troubles ...