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The New England Journal of Medicine|September 25, 2024
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia BAdam Cuker, Kaan Kavakli, Laurent Frenzel, et al.Research and Practice in Thrombosis and Haemostasis|December 17, 2024
Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trialAndrew D Leavitt, Johnny Mahlangu, Priyanka Raheja, et al.Frontiers in Immunology|August 18, 2025
New insights into Wiskott-Aldrich syndrome: ten novel WAS mutations and their clinical impact in a Brazilian cohortLucas W Santos, Samuel S Medina, Jéssica O Frade-Guanaes, et al.Journal of Thrombosis and Haemostasis : JTH|April 13, 2024
Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia ABella Madan, Margareth C Ozelo, Priyanka Raheja, et al.Human Gene Therapy|February 14, 2022
Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia ARobert Klamroth, Gregory Hayes, Tatiana Andreeva, et al.Journal of Thrombosis and Haemostasis : JTH|September 7, 2023
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1Brian O'Mahony, Amy L Dunn, Andrew D Leavitt, et al.The New England Journal of Medicine|February 22, 2023
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia AJohnny Mahlangu, Radoslaw Kaczmarek, Annette von Drygalski, et al.The New England Journal of Medicine|December 6, 2013
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia BJerry S Powell, K John Pasi, Margaret V Ragni, et al.Nature Medicine|February 14, 2006
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune responseCatherine S Manno, Glenn F Pierce, Valder R Arruda, et al.Pageof 7