Muscular Dystrophies, Limb-Girdle

Untangling Sarepta’s Gene Therapy Fallout and Trust Deficit

1 month ago talkbio0Tagged AAVrh74 vector, clinical holds, ELEVIDYS, gene therapy, Muscular Dystrophies, Limb-Girdle, Muscular Dystrophy, Duchenne, patient deaths, Regulatory Action, Translucent, United States Food and Drug Administration

Sarepta Therapeutics; gene therapy; Elevidys; Duchenne muscular dystrophy; limb-girdle muscular dystrophy; patient deaths; FDA; AAVrh74 vector; regulatory action; clinical holds; transparency

Lack of Transparency Tarnishes Sarepta’s Sheen as Patient Deaths Trigger FDA Battle

2 months ago talkbio0Tagged ELEVIDYS, gene therapy, lack of transparency, Market, Muscular Dystrophies, Limb-Girdle, Muscular Dystrophy, Duchenne, patient deaths, Regulatory Action, United States Food and Drug Administration, Withdraw (activity)

Sarepta Therapeutics; Elevidys; patient deaths; FDA; lack of transparency; gene therapy; market withdrawal; regulatory action; limb-girdle muscular dystrophy (LGMD); Duchenne muscular dystrophy

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

2 months 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

Sarepta’s rAAVrh74 Viral Vector Receives FDA Platform Technology Designation for Gene Therapy

3 months ago talkbio0Tagged gene therapy, Infrequent, Muscular Dystrophies, Limb-Girdle, platform technology designation, SRP-9003, United States Food and Drug Administration, Viral Vector

Sarepta Therapeutics; FDA; platform technology designation; rAAVrh74 viral vector; gene therapy; SRP-9003; limb-girdle muscular dystrophy; rare diseases