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PO045 | FROM PROPHYLAXIS TO ENDOGENOUS CONTROL: A SIX-MONTH REAL-WORLD EXPERIENCE WITH VALOCTOCOGENE ROXAPARVOVEC GENE THERAPY M. Basso¹, A. Ferretti¹, M. Sacco¹, S. Lancellotti¹, T. Pirozzi¹, L. Di Gennaro¹, R. De Cristofaro^1|2 | ¹Servizio Malattie Emorragiche e Trombotiche, Fondazione Policlinico Universitario Gemelli IRCCS, Italy; ²Dipartimento di Medicina e Chirurgia Traslazionale, Università Cattolica S. Cuore, Roma, Italy

Raimondo De Cristofaro

26-10-2026

https://doi.org/10.4081/btvb.2026.861

25

PDF: 13
The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art

Giovanni Di Minno, Gaia Spadarella, Ilenia Lorenza Calcaterra, Giancarlo Castaman, Paolo Simioni, Raimondo De Cristofaro, Cristina Santoro, Flora Peyvandi, Matteo Di Minno

04-09-2024

https://doi.org/10.4081/btvb.2024.144

2091

PDF: 359

SUPPLEMENTARY MATERIAL: 218

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