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

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

Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Elsevier 2020-11-01
Series:Hematology, Transfusion and Cell Therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2531137920303850
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spelling doaj-5092e78d892c4dc8a80837237b6fc4c42020-11-25T03:05:41ZengElsevierHematology, Transfusion and Cell Therapy2531-13792020-11-014260A FIRST-IN-HUMAN FOUR-YEAR FOLLOW-UP STUDY OF DURABLE THERAPEUTIC EFFICACY AND SAFETY OF AAV GENE THERAPY WITH VALOCTOCOGENE ROXAPARVOVEC FOR SEVERE HEMOPHILIA AM.C. Ozelo0K.J. Pasi1S. Rangarajan2N. Mitchell3W. Lester4E. Symington5B. Madan6M. Laffan7C.B. Russell8M. Li9B. Kim10G.F. Pierce11W.Y. Wong12Centro de Hematologia e Hemoterapia (Hemocentro), Universidade Estadual de Campinas (UNICAMP), Campinas, SP, BrazilBarts and the London School of Medicine and Dentistry, London, United KingdomUniversity Hospital Southampton, Southampton, United KingdomBioMarin Pharmaceutical Inc., Novato, United StatesUniversity Hospitals Birmingham NHS Foundation Trust, Birmingham, United KingdomCambridge University Hospitals NHS Foundation Trust, Cambridge, United KingdomGuy's & St. Thomas’NHS Foundation Trust, London, United KingdomCentre for Haematology, Imperial College London, London, United KingdomBioMarin Pharmaceutical Inc., Novato, United StatesBioMarin Pharmaceutical Inc., Novato, United StatesBioMarin Pharmaceutical Inc., Novato, United StatesConsultant, La Jolla, United StatesBioMarin Pharmaceutical Inc., Novato, United Stateshttp://www.sciencedirect.com/science/article/pii/S2531137920303850
collection DOAJ
language English
format Article
sources DOAJ
author 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
spellingShingle 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
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
Hematology, Transfusion and Cell Therapy
author_facet 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
author_sort M.C. Ozelo
title 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
title_short 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
title_full 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
title_fullStr 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
title_full_unstemmed 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
title_sort 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
publisher Elsevier
series Hematology, Transfusion and Cell Therapy
issn 2531-1379
publishDate 2020-11-01
url http://www.sciencedirect.com/science/article/pii/S2531137920303850
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