BLEEDING, FVIII ACTIVITY, AND SAFETY 3 YEARS AFTER GENE TRANSFER WITH VALOCTOCOGENE ROXAPARVOVEC: RESULTS FROM GENER8-1
MC Ozeloa, J Mahlangub, B Madanc, J Masond,e, F Peyvandif,g, AV Drygalskih, G Yamaguti-Hayakawaa, TM Robinsoni, SW Pipej, TT Gener-Eighk
a Department of Internal Medicine, Faculdade de Ciências Médicas (FCM), Centro de Hematologia e Hemoterapia (Hemocentro), Universidade Estadual de Campinas (UNICAMP), Campinas, Brazil
b Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa
c Guy's & St. Thomas'NHS Foundation Trust, London, UK
d University of Queensland, Brisbane, Australia
e Queensland Haemophilia Centre, Cancer Care Services, Royal Brisbane and Women's Hospital, Brisbane, Australia
f Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy
g Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy
h Department of Medicine, University of California San Diego, San Diego, United States
i BioMarin Pharmaceutical, Inc., Novato, United States
j Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, United States
k GENEr8-1 Trial Centers
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