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A FIRST-IN-HUMAN FOUR-YEAR FOLLOW-UP STUDY OF DURABLE THERAPEUTIC EFFICACY AND SAFETY OF AAV GENE THERAPY WITH VALOCTOCOGENE ROXAPARVOVEC FOR SEVERE HEMOPHILIA A
M.C. Ozeloa, K.J. Pasib, S. Rangarajanc, N. Mitchelld, W. Lestere, E. Symingtonf, B. Madang, M. Laffanh, C.B. Russelld, M. Lid, B. Kimd, G.F. Piercei, W.Y. Wongd
a Centro de Hematologia e Hemoterapia (Hemocentro), Universidade Estadual de Campinas (UNICAMP), Campinas, SP, Brazil
b Barts and the London School of Medicine and Dentistry, London, United Kingdom
c University Hospital Southampton, Southampton, United Kingdom
d BioMarin Pharmaceutical Inc., Novato, United States
e University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom
f Cambridge University Hospitals NHS Foundation Trust, Cambridge, United Kingdom
g Guy's & St. Thomas’NHS Foundation Trust, London, United Kingdom
h Centre for Haematology, Imperial College London, London, United Kingdom
i Consultant, La Jolla, United States
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