Liver Failure, Acute
Sarepta Therapeutics Faces FDA Crackdown after Third Patient Death; George Tidmarsh Named Top FDA Drug Regulator; Multiple Drugs Rejected; Pharma Invests Billions in US Manufacturing
Sarepta Therapeutics; FDA; gene therapy; Elevidys; acute liver failure; clinical hold; George Tidmarsh; drug rejection; reshoring; pharmaceutical manufacturing
Sarepta Reports Third Patient Death in Gene Therapy Studies for Limb-Girdle Muscular Dystrophy
Sarepta Therapeutics; limb-girdle muscular dystrophy; SRP-9004; gene therapy; patient death; acute liver failure; FDA clinical hold; AAV vector; Elevidys; safety concerns
FDA Investigates Patient Deaths Linked to Sarepta’s Elevidys Gene Therapy for Duchenne Muscular Dystrophy
FDA; Sarepta; Elevidys; Duchenne muscular dystrophy; gene therapy; patient deaths; acute liver failure; liver toxicity; regulatory investigation; non-ambulatory patients
Second Patient Dies After Receiving Sarepta’s Elevidys Gene Therapy for DMD
Duchenne muscular dystrophy; gene therapy; Sarepta Therapeutics; Elevidys; acute liver failure; AAV-based gene therapy; patient death; non-ambulatory patients; FDA; risk mitigation
Sarepta Halts Duchenne Gene Therapy in Some Patients After Second Death
Sarepta Therapeutics; gene therapy; Duchenne muscular dystrophy; Elevidys; acute liver failure; non-ambulatory patients; FDA; clinical trial pause; immunosuppression; patient safety