The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art

Despite major advances in prophylaxis, no repeated dosing regimen with currently employed extended-half-life or non-factor products replaces the advantages of a long-term cure in persons with severe congenital hemophilia A and B (HA, HB). They indeed live with the risk of breakthrough bleedings, an...

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Bibliographic Details

Published in:	Bleeding, Thrombosis and Vascular Biology
Main Authors:	Giovanni Di Minno, Gaia Spadarella, Ilenia Lorenza Calcaterra, Giancarlo Castaman, Paolo Simioni, Raimondo De Cristofaro, Cristina Santoro, Flora Peyvandi, Matteo Di Minno
Format:	Article
Language:	English
Published:	PAGEPress Publications 2024-09-01
Subjects:	Adeno-associated virus gene therapy person-reported outcomes laboratory monitoring emicizumab extended half-life products
Online Access:	https://www.btvb.org/btvb/article/view/144