Liver Failure, Acute

FDA Investigates Patient Deaths Linked to Sarepta’s Elevidys Gene Therapy for Duchenne Muscular Dystrophy

5 days ago talkbio0Tagged ELEVIDYS, Evaluation, gene therapy, Liver, Liver Failure, Acute, Muscular Dystrophy, Duchenne, patient deaths, regulatory, Toxicity aspects, Unable to walk (finding), United States Food and Drug Administration

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

2 weeks ago talkbio0Tagged AAV-based gene therapy, ELEVIDYS, gene therapy, Liver Failure, Acute, Muscular Dystrophy, Duchenne, Patient Death, risk mitigation, Unable to walk (finding), United States Food and Drug Administration

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

2 weeks ago talkbio0Tagged Clinical Trials, ELEVIDYS, gene therapy, Liver Failure, Acute, Muscular Dystrophy, Duchenne, patient safety, Therapeutic immunosuppression, Unable to walk (finding), United States Food and Drug Administration

Sarepta Therapeutics; gene therapy; Duchenne muscular dystrophy; Elevidys; acute liver failure; non-ambulatory patients; FDA; clinical trial pause; immunosuppression; patient safety