Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
Steven W PipeFrank W G LeebeekMichael RechtNigel S KeyGiancarlo CastamanWolfgang A MiesbachSusan LattimoreKathelijne PeerlinckPaul Van der ValkMichiel CoppensPeter KampmannKarina MeijerNiamh O'ConnellK John PasiDaniel P HartRashid KazmiJan AstermarkCedric R J R HermansRobert KlamrothRichard LemonsNathan VisweshwarAnnette von DrygalskiGuy YoungShelley E CraryMiguel EscobarEsteban GomezRebecca Kruse-JarresDoris V QuonEmily SymingtonMichael WangAllison P WheelerRobert GutYing P LiuRicardo E DolmetschDavid L CooperYanyan LiBrahm GoldsteinPaul E MonahanPublished in: The New England journal of medicine (2023)
Etranacogene dezaparvovec gene therapy was superior to prophylactic factor IX with respect to the annualized bleeding rate, and it had a favorable safety profile. (Funded by uniQure and CSL Behring; HOPE-B ClinicalTrials.gov number, NCT03569891.).
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