Hematology, Transfusion and Cell Therapy (Nov 2020)
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
- Journal volume & issue
-
Vol. 42
p. 60