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Article

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

22 Feb 2023

Abstract excerpt

BACKGROUND: Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby protecting against bleeding without burdensome factor IX replacement. METHODS: genome copies per kilogram of body weight) to 54 men with hemophilia B (factor IX activity ≤2% of the normal value)...

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Share to Blood Coagulation and Thrombosis Mechanisms
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PMID: 36812434
DOI: 10.1056/nejmoa2211644

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