Sarepta Therapeutics is facing mounting challenges surrounding its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys. After three patient deaths linked to liver failure, including a case related to another Sarepta gene therapy trial, the FDA formally requested a voluntary halt on Elevidys shipments—a request Sarepta initially refused. Subsequently, Sarepta reversed course, agreeing to temporarily pause shipments to maintain a productive relationship with the FDA. The agency has threatened to use its full regulatory authority to enforce compliance. Meanwhile, Sarepta’s regulatory standing has weakened as it lost its gene therapy platform designation and clinical trials for limb-girdle muscular dystrophy (LGMD) also face holds amid safety concerns. The episode underscores tensions over safety transparency and regulatory standards for novel gene therapies.