Sarepta Therapeutics faces heightened scrutiny following a second death due to acute liver failure associated with its gene therapy Elevidys, approved for Duchenne muscular dystrophy. Both fatalities occurred in non-ambulatory patients, spurring sharp declines in Sarepta’s stock and industry concern. The company has proposed immunosuppressive regimens to mitigate liver toxicity, with regulatory responses pending. The fatalities underscore ongoing challenges in gene therapy safety monitoring and risk management for rare pediatric diseases.