Sarepta Therapeutics has temporarily halted all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, following mounting safety concerns and an FDA request. The pause follows three patient deaths linked to acute liver failure, two of which were associated with Elevidys, and a third involving a related limb-girdle muscular dystrophy gene therapy trial. After initial refusal to comply with FDA's halt request, Sarepta reversed course to maintain a cooperative relationship and address the agency's demands, including revising Elevidys' safety labeling. The FDA has also placed clinical holds on Sarepta's LGMD trials and revoked the platform technology designation previously granted to the company's gene therapy approach, underscoring regulatory scrutiny amid patient safety risks. This situation exemplifies the challenging balance between innovation and safety in gene therapy development.