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. Ozelo,
• K.J. Pasi,
• S. Rangarajan,
• N. Mitchell,
• W. Lester,
• E. Symington,
• B. Madan,
• M. Laffan,
• C.B. Russell,
• M. Li,
• B. Kim,
• G.F. Pierce,
• W.Y. Wong

Affiliations

M.C. Ozelo

Centro de Hematologia e Hemoterapia (Hemocentro), Universidade Estadual de Campinas (UNICAMP), Campinas, SP, Brazil

K.J. Pasi

Barts and the London School of Medicine and Dentistry, London, United Kingdom

S. Rangarajan

University Hospital Southampton, Southampton, United Kingdom

N. Mitchell

BioMarin Pharmaceutical Inc., Novato, United States

W. Lester

University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom

E. Symington

Cambridge University Hospitals NHS Foundation Trust, Cambridge, United Kingdom

B. Madan

Guy's & St. Thomas’NHS Foundation Trust, London, United Kingdom

M. Laffan

Centre for Haematology, Imperial College London, London, United Kingdom

C.B. Russell

BioMarin Pharmaceutical Inc., Novato, United States

M. Li

BioMarin Pharmaceutical Inc., Novato, United States

B. Kim

BioMarin Pharmaceutical Inc., Novato, United States

G.F. Pierce

Consultant, La Jolla, United States

W.Y. Wong

BioMarin Pharmaceutical Inc., Novato, United States