EFFICACY AND SAFETY OF VALOCTOCOGENE ROXAPARVOVEC 4 YEARS AFTER GENE TRANSFER IN GENER8-1
MC Ozeloa, AD Leavittb, J Mahlanguc, G Kenetd, SW Pipee, S Chouf, R Klamrothg,h, F Peyvandii,j, T Robinsonk, GT Groupl
a Centro de Hematologia e Hemoterapia (Hemocentro), Department of Internal Medicine Faculdade de Ciências Médicas (FCM), Universidade Estadual de Campinas (UNICAMP), Campinas, SP, Brazil
b Adult Hemophilia Treatment Center, University of California San Francisco, San Francisco, CA, USA
c Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa
d The National Hemophilia Center and Amalia Biron Research Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel Hashomer, Tel Aviv University, Tel Aviv, Israel
e Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI, USA
f Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan
g Vascular Medicine and Haemostaseology, Vivantes Klinikum im Friedrichshain, Berlin, Germany
h Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany
i Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy
j Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy
k BioMarin Pharmaceutical Inc., Novato, CA, USA
l GENEr8-1 Trial Group
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