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LB 01.3 : Relationship between transgene-produced FVIII and bleeding rates 2 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

Researchers

Presenter

  • J. Mahlangu

Principal Investigators

  • S. Chou

  • A. Dunn

  • A. von Drygalski

  • R. Kaczmarek

  • G. Kenet

  • M. Laffan

  • A. Leavitt

  • B. Madan

  • J. Mason

  • J. Oldenburg

  • M. Ozelo

  • F. Peyvandi

  • D. Quon

  • M. Reding

  • S. Shapiro

  • H. Yu

  • T. Robinson

  • S. Pipe

Medical Centers

  • University of the Witwatersrand, National Health Laboratory Service and Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, Gauteng, South Africa

  • APHM, Department of Pediatric Hematology Oncology, La Timone Children’s Hospital & Aix Marseille Univ, INSERM, INRA, C2VN, Marseille, Provence-Alpes-Cote d'Azur, France

  • Division of Oncology/Hematology and Internal Medicine,National Taiwan University Hospital,Taipei,Taiwan, Province of China

  • Nationwide Children's Hospital Division of Hematology, Oncology, and Bone Marrow Transplant and The Ohio State University College of Medicine, Columbus, Ohio, United States

  • Division of Gastroenterology, Dept of Medicine, University of California, San Diego, San Diego, California, United States

  • Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, and Hirszfeld Institute of Immunology and Experimental Therapy, Indianapolis, Indiana, United States

  • Sheba medical center& Tel Aviv University, Tel Aviv, Tel Aviv, Israel

  • Centre for Haematology, Imperial Collegel, London, United Kingdom

  • University of California, San Francisco, San Francisco, California, United States

  • Guy's And St Thomas NHS Foundation Trust, London, United Kingdom

  • Queensland Haemophilia Centre, Royal Brisbane and Women's Hospital, and University of Queensland, Brisbane, Queensland, Australia

  • Institute of Experimental Hematology and Transfusion Medicine and Centre for Rare Diseases, Universitätsklinikum Bonn, Bonn, Nordrhein-Westfalen, Germany

  • Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Sao Paulo, Brazil

  • Fondazione IRCCS Ca’ Granda - Ospedale Maggiore Policlinico, UOC Medicina Generale, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, and Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Lombardia, Italy

  • Orthopaedic Hemophilia Treatment Center, Los Angeles, California, United States

  • Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, Minnesota, United States

  • Oxford University Hospitals National Health Service Foundation Trust, University of Oxford, and Oxford National Institute for Health Research Biomedical Research Centre, Oxford, England, United Kingdom

  • BioMarin Pharmaceutical Inc., Novato, United States

  • Department of Gastroenterology and Hepatology, University of Michigan, Ann Arbor, Michigan, United States

Locations

  • South Africa

  • France

  • Republic of South Korea

  • United States

  • Israel

  • United Kingdom

  • Australia

  • Germany

  • Brazil

  • Italy

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Hemophilia

Biomarkers

  • N/A

Drug/Treatment

  • FVIII

Outcome

  • N/A

Study Design

  • Open Label

  • Non Interventional

  • Multicenteric

Phase

  • III

Study Id's

  • NCT03370913

Sponsors

  • N/A

Result

  • N/A

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