Sarepta Therapeutics faces intensified scrutiny after the FDA requested the halt of its Duchenne muscular dystrophy gene therapy, Elevidys, following two patient deaths from acute liver failure. Sarepta also reported a third death in its limb-girdle muscular dystrophy gene therapy trial involving a 51-year-old participant, raising broader safety concerns around its gene therapy platform. These events prompted Sarepta to pause shipments for non-ambulatory Duchenne patients and add a black box warning of potential liver injury. Despite criticism over limited efficacy data and internal dissent within the FDA at the time of Elevidys' accelerated approval, Sarepta defends its transparency amid these setbacks. The company is undergoing a significant organizational restructuring, including a 36% workforce reduction, to conserve $400 million annually and refocus on its pipeline, including siRNA programs. This turmoil has impacted Sarepta's stock volatility and the commercial viability of Elevidys.