vector
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
Neurogene’s Rett Syndrome Gene Therapy Shows Efficacy but Raises Safety Concerns
Neurogene, Rett syndrome, gene therapy, NGN-401, AAV vector, clinical trial, safety concerns, efficacy results