Sarepta Therapeutics is confronting regulatory challenges after the FDA decided to request a halt on shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, following the deaths of two teenage patients from acute liver failure. The FDA is also investigating the safety profile of Elevidys, and the company has reported a third death of a 51-year-old man involved in a limb-girdle muscular dystrophy gene therapy trial. Sarepta has responded by pausing new shipments to non-ambulatory patients and plans to include a black box warning on Elevidys for potential acute liver injury. This regulatory scrutiny coincides with Sarepta's announcement of a major company restructuring involving a 36% workforce reduction to secure financial stability. Sarepta CEO Douglas Ingram emphasized transparency and the need for continued progress despite setbacks, amid ongoing debates within the industry regarding the risk-benefit profile of AAV-based gene therapies.