Relasyon sa pagitan ng Transgene-Produced FVIII at Bleeding Rate 2 Taon Pagkatapos ng Gene Transfer Sa Valoctocogene Roxaparvovec: Mga Resulta Mula sa Gener8-1
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Relasyon sa pagitan ng Transgene-Produced FVIII at Bleeding Rate 2 Taon Pagkatapos ng Gene Transfer Sa Valoctocogene Roxaparvovec: Mga Resulta Mula sa Gener8-1

Iniharap ni: Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, Faculty of the Health Sciences-University of the Witwatersrand at National Health Laboratory Service, Johannesburg, South Africa

J. Mahlangu1, H. Chambost2, S. Chou3, A. Dunn4, A. von Drygalski5, R. Kaczmarek6, G. Kenet7, M. Laffan8, A. Leavitt9, B. Madan10, J. Mason11, J. Oldenburg12, M. Ozelo13, F. Peyvandi14, D. Quon15, M. Reding16, S. Shapiro17, H. Yu18, T. Robinson18, S. Pipe19

1University of the Witwatersrand, National Health Laboratory Service at Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, Gauteng, South Africa
2APHM, Department of Pediatric Hematology Oncology, La Timone Children's Hospital at Aix Marseille Univ, INSERM, INRA, C2VN, Marseille, Provence-Alpes-Cote d'Azur, France
3Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Keelung, Taiwan (Republic of China)
4Nationwide Children's Hospital Division of Hematology, Oncology, and Bone Marrow Transplant at The Ohio State University College of Medicine, Columbus, Ohio, United States
5Dibisyon ng Hematology/Oncology, Kagawaran ng Medisina, Unibersidad ng California San Diego, San Diego, California, Estados Unidos
6Indiana University School of Medicine, IUPUI-Wells Center para sa Pediatric Research, at Hirszfeld Institute of Immunology and Experimental Therapy, Indianapolis, Indiana, United States
7Sheba medical center at Tel Aviv University, Tel Aviv, Tel Aviv, Israel
8Center for Hematology, Imperial College London, London, England, United Kingdom
9Unibersidad ng California San Francisco, San Francisco, California, Estados Unidos
10Guy's & St. Thomas' NHS Foundation Trust, London, England, United Kingdom
11Queensland Haemophilia Center, Royal Brisbane at Women's Hospital, at University of Queensland, Brisbane, Queensland, Australia
12Institute of Experimental Hematology and Transfusion Medicine at Center for Rare Diseases, Universitätsklinikum Bonn, Bonn, Nordrhein-Westfalen, Germany
13Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Sao Paulo, Brazil
14Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, UOC Medicina Generale, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center at Fondazione Luigi Villa, at Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Lombardia, Italy
15Orthopedic Hemophilia Treatment Center, Los Angeles, California, United States
16Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, Minnesota, United States
17Oxford University Hospitals National Health Service Foundation Trust, University of Oxford, at Oxford National Institute for Health Research Biomedical Research Center, Oxford, England, United Kingdom
18BioMarin Pharmaceutical, Inc., Novato, California, United States

19Mga Departamento ng Pediatrics at Patolohiya, Unibersidad ng Michigan, Ann Arbor, Michigan, Estados Unidos

Mga Pangunahing Mga Punto ng Data

Valoctocogene Roxaparvovec Buod ng Kaligtasan para sa Taon 2
Annualized Treated Bleeding Rate/FVIII Utilization

Ang mga resulta mula sa phase 3 GENEr8-1 na pag-aaral sa mga pasyenteng may hemophilia A na ginagamot sa valoctocogene roxaparvovec ay nagpapakita ng pare-parehong pagbawas sa mga ginagamot na pagdugo/taon at taunang paggamit ng FVIII mula sa taong 1 hanggang sa taong 2 kumpara sa baseline. Ang mga baseline na halaga ay mula sa 6 na buwang prospective na panahon ng pangongolekta ng data sa noninterventional study BMN 270-902.

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Sinusuportahan ng mga grant na pang-edukasyon mula sa Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, at uniQure, Inc.

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