First conditional marketing authorization approval in the European Union for hemophilia “A” gene therapy
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference7 articles.
1. Haemophilia gene therapy: from trailblazer to gamechanger. Haemophilia;Evens;Haemophilia.,2018
2. Valoctocogene roxaparvovec gene therapy for hemophilia A;Ozelo;N. Engl. J. Med.,2022
3. Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A;Pasi;Haemophilia.,2021
4. Multiyear factor VIII expression after AAV gene transfer for hemophilia A;George;N. Engl. J. Med.,2021
5. IL-15 blockade and rapamycin rescue multifactorial loss of factor VIII from AAV-transduced hepatocytes in hemophilia A mice;Butterfield;Mol. Ther.,2022
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