Sarepta Therapeutics has temporarily halted shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, following safety concerns that include three patient deaths tied to acute liver failure. After initially refusing an FDA request to pause shipments, Sarepta reversed course to maintain a constructive relationship with regulators. The FDA has expressed deep disappointment and threatened full regulatory action. Safety investigations and labeling revisions are ongoing amid concerns about the therapy's risk profile and clinical evidence robustness.