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Roctavian gene therapy for hemophilia A

11 Jul 2024

Abstract excerpt

ABSTRACT: After successful efforts in adeno-associated virus (AAV) gene addition for hemophilia B gene therapy, the development of valoctocogene roxaparvovec (Roctavian; Biomarin) over the past decade represents a potential new hemophilia A (HA) treatment paradigm. Roctavian is the first licensed HA gene therapy that was conditionally approved in Europe in August 2022 and approved in the United States in June...

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PMID: 38991118
DOI: 10.1182/bloodadvances.2023011847

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