Sarepta Therapeutics is facing intensified regulatory and commercial challenges following the confirmation of a third patient death related to its gene therapy products. The newest fatality involved a 51-year-old participant in a clinical trial for limb-girdle muscular dystrophy, who died from acute liver failure, joining two prior deaths from Elevidys, Sarepta’s treatment for Duchenne muscular dystrophy. The FDA has formally requested Sarepta halt shipments of Elevidys, previously paused for non-ambulatory patients after two teenage deaths, and is considering withdrawing the therapy from the market entirely. These developments underline growing safety concerns with AAV-mediated gene therapies and have led to internal leadership changes and strategic restructuring within Sarepta, including staff reductions and executive departures. The gene therapy approvals have been controversial, with FDA reviewers initially recommending rejection and concerns over limited efficacy data. Sarepta executives have emphasized transparency while managing the crisis, facing criticism from financial analysts and patient advocates alike.