Sarepta Therapeutics said it has temporarily suspended shipments of Elevidys® (delandistrogene moxeparvovec-rokl) for infusion in non-ambulatory patients following the second death in three months of ...

Sarepta Therapeutics ( NASDAQ: SRPT) reported a second fatality from acute liver failure in a patient treated with its gene ...

Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for ...

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...