Patient Death – Talk Bio

Sarepta Reports Third Patient Death in Gene Therapy Studies for Limb-Girdle Muscular Dystrophy

6 days ago talkbio0Tagged AAVS1 gene, ELEVIDYS, gene therapy, Liver Failure, Acute, Muscular Dystrophies, Limb-Girdle, Patient Death, Safety, Study on Hold, United States Food and Drug Administration, vector

Sarepta Therapeutics; limb-girdle muscular dystrophy; SRP-9004; gene therapy; patient death; acute liver failure; FDA clinical hold; AAV vector; Elevidys; safety concerns

Second Patient Dies After Receiving Sarepta’s Elevidys Gene Therapy for DMD

1 month 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

Patient Death in Rocket Pharmaceuticals Gene Therapy Trial Prompts FDA Clinical Hold

2 months ago talkbio0Tagged acute systemic infection, Adverse event, Capillary Leak Syndrome, gene therapy, Glycogen Storage Disease Type IIb, Patient Death, Rocket, RP-A501, Study on Hold, United States Food and Drug Administration

Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; FDA; patient death; capillary leak syndrome; acute systemic infection; RP-A501; adverse event