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Article

Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B

25 Sept 2024

Abstract excerpt

BACKGROUND: Fidanacogene elaparvovec, an adeno-associated virus (AAV) gene-therapy vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX-Padua), was associated with sustained factor IX activity in a phase 1-2a study. METHODS: vector genome copies per kilogram of body weight. Men 18 to 65 years of age with hemophilia B and a factor IX level of 2% or less were eligible for...

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PMID: 39321362
DOI: 10.1056/nejmoa2302982

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