The FDA has reversed its earlier halt on Sarepta Therapeutics’ gene therapy Elevidys for Duchenne muscular dystrophy, allowing shipments to ambulatory patients to resume after investigations found the death of an 8-year-old patient unrelated to the treatment. The ongoing controversy involves previous deaths tied to the therapy and safety concerns, leading to a paused distribution for non-ambulatory patients. The agency and company continue safety label updates and risk mitigation discussions for this vulnerable group, underscoring the complex regulatory landscape for advanced gene therapies.