Sarepta Therapeutics has temporarily paused shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy (DMD), following an escalating standoff with the FDA. The FDA requested a halt after three patient deaths linked to liver failure were reported, including one from a separate Sarepta gene therapy trial for limb-girdle muscular dystrophy. Sarepta initially resisted but reversed course to maintain regulatory collaboration. Concurrently, the FDA has imposed clinical holds on Sarepta’s limb-girdle muscular dystrophy studies and revoked the company's gene therapy platform designation due to safety concerns. The company is working with the FDA on safety labeling updates for Elevidys.