Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table-Reference-Cited by-同舟云学术

Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table

Published:2020-03-23 Issue:3 Volume:26 Page:443-449
ISSN:1351-8216
Container-title:Haemophilia
language:en
Short-container-title:Haemophilia

Author:

Pierce Glenn F.¹^ORCID,Pasi K. John²^ORCID,Coffin Donna¹^ORCID,Kaczmarek Radoslaw³⁴,Lillicrap David⁵,Mahlangu Johnny⁶^ORCID,Rottellini Dawn⁷,Sannié Thomas⁸,Srivastava Alok⁹^ORCID,VandenDriessche Thierry¹⁰¹¹,Weill Alain¹^ORCID,Almeida Antonio J.,Baumann Alain,Brooker Mark,Durante Luisa,Naccache Mayss,Robinson Fiona,

Affiliation:

1. World Federation of Hemophilia Montreal QC Canada

2. Royal London Haemophilia Centre Barts and The London School of Medicine and Dentistry London UK

3. Department of Pediatrics Indiana University School of Medicine Indianapolis IN USA

4. Laboratory of Glycobiology Hirszfeld Institute of Immunology and Experimental Therapy Wroclaw Poland

5. Department of Pathology & Molecular Medicine Queen's University Kingston ON Canada

6. Haemophilia Comprehensive Care Centre NHLS and Charlotte Maxeke Johannesburg Academic Hospital University of the Witwatersrand Johannesburg South Africa

7. National Hemophilia Foundation New York NY USA

8. French Haemophilia Association (AFH) Paris France

9. Department of Haematology Christian Medical College Vellore Vellore India

10. Department of Gene Therapy & Regenerative Medicine Vrije Universiteit Brussel (VUB) Brussels Belgium

11. Department of Cardiovascular Sciences Center for Molecular & Vascular Biology University of Leuven Leuven Belgium

Publisher

Wiley

Subject

Genetics (clinical),Hematology,General Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/hae.13971

Reference23 articles.

1. Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes

2. Gene therapy for hemophilia

3. BioMarin Pharmaceutical Inc.BioMarin Submits Marketing Authorization Application to European Medicines Agency for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A.https://investors.biomarin.com/2019‐11‐21‐BioMarin‐Submits‐Marketing‐Authorization‐Application‐to‐European‐Medicines‐Agency‐for‐Valoctocogene‐Roxaparvovec‐to‐Treat‐Severe‐Hemophilia‐A. Accessed January 08 2020.

4. BioMarin Pharmaceutical Inc.BioMarin Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A.https://investors.biomarin.com/2019‐12‐23‐BioMarin‐Submits‐Biologics‐License‐Application‐to‐U‐S‐Food‐and‐Drug‐Administration‐for‐Valoctocogene‐Roxaparvovec‐to‐Treat‐Hemophilia‐A. Accessed January 08 2020.

5. Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

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