Sarepta Therapeutics faces mounting challenges as the FDA prepares to request a halt to shipments of its gene therapy Elevidys for Duchenne muscular dystrophy. This decision follows the deaths of multiple patients from acute liver failure, including two teenagers treated with Elevidys and a 51-year-old non-ambulatory man receiving an experimental limb-girdle muscular dystrophy gene therapy. Sarepta's controversial approvals, particularly for Elevidys, had faced internal FDA opposition and skepticism over efficacy. The FDA's investigation reflects ongoing safety concerns amid calls for greater transparency and industry-wide reassessment of AAV-based gene therapies. Sarepta’s CEO defended the company's transparency amid criticism over delayed disclosure of patient deaths. Industry voices debate regulatory responses, with former FDA officials supporting the pause in shipments amid the evolving crisis.