Sarepta Therapeutics is facing escalating safety concerns as a third patient has died after receiving one of its gene therapies. The latest fatality involved a 51-year-old participant in a limb-girdle muscular dystrophy clinical trial who died from acute liver failure. Previously, two teenage patients treated with Sarepta's Elevidys gene therapy for Duchenne muscular dystrophy also died from liver complications. These incidents have prompted the FDA to request Sarepta halt shipments of Elevidys, especially for older patients, triggering a formal investigation into liver risks associated with the therapy. Sarepta CEO Douglas Ingram has defended the company's transparency despite criticism over delayed disclosures. Industry experts, including former FDA CBER chief Peter Marks, have supported suspension of the therapy shipments, citing patient safety. This crisis spotlights challenges in AAV-vectored gene therapies balancing efficacy and safety in rare genetic diseases.