Following two patient deaths linked to acute liver failure shortly after treatment with Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys, the FDA has begun reviewing the therapy's safety profile. Sarepta has implemented safety measures including a proposed enhanced immunosuppression regimen and has suspended shipments for non-ambulatory patients. Regulatory scrutiny remains focused on assessing further necessary actions as concern grows regarding adverse hepatic effects associated with AAV-based gene therapies.