Sarepta Therapeutics has temporarily halted shipments of its Duchenne muscular dystrophy gene therapy Elevidys following safety concerns tied to three patient deaths linked to acute liver failure. The FDA requested the voluntary suspension after the third fatality, related to a separate Sarepta therapy using the same viral vector. This marks a regulatory escalation amid disputes between Sarepta and the agency, which has expressed deep disappointment over Sarepta’s initial refusal to halt shipments. The FDA also placed holds on Sarepta’s limb-girdle muscular dystrophy trials and revoked its gene therapy platform designation. The crisis underscores challenges in overseeing novel gene therapies with serious safety risks.