Relasyon ant transjèn FVIII ki pwodui ak pousantaj senyen 2 ane apre transfè jèn ak Valoctocogene Roxaparvovec: Rezilta ki soti nan Jener8-1
Pwen esansyèl Soti nan 30yèm Kongrè ISTH la

Relasyon ant transjèn FVIII ki pwodui ak pousantaj senyen 2 ane apre transfè jèn ak Valoctocogene Roxaparvovec: Rezilta ki soti nan Jener8-1

Prezante pa: Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, Fakilte Syans Sante-University of Witwatersrand ak Sèvis Laboratwa Sante Nasyonal, Johannesburg, Lafrik di sid.

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. Tiyo19

1University of the Witwatersrand, Sèvis Laboratwa Sante Nasyonal ak Sant Swen Sante Emofili, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, Gauteng, Lafrik di sid
2APHM, Depatman nkoloji ematoloji pedyat, Lopital Timoun La Timone & Aix Marseille Univ, INSERM, INRA, C2VN, Marseille, Provence-Alpes-Cote d'Azur, Frans
3Divizyon ematoloji, Depatman Medsin Entèn, National Taiwan University Hospital, Taipei, Keelung, Taiwan (Repiblik Lachin)
4Nationwide Children's Hospital Division of Hematology, Oncology, and Bone Marrow Transplant ak The Ohio State University College of Medicine, Columbus, Ohio, Etazini.
5Divizyon ematoloji/nkoloji, Depatman Medsin, University of California San Diego, San Diego, Kalifòni, Etazini
6Indiana University School of Medicine, IUPUI-Wells Center for Pedyat Research, ak Hirszfeld Institute of Immunology and Experimental Therapy, Indianapolis, Indiana, Etazini.
7Sheba Medical Center & Tel Aviv University, Tel Aviv, Tel Aviv, Israel
8Sant pou ematoloji, Imperial College London, London, Angletè, Wayòm Ini
9University of California San Francisco, San Francisco, Kalifòni, Etazini
10Guy's & St. Thomas' NHS Foundation Trust, London, Angletè, Wayòm Ini
11Queensland Haemophilia Centre, Royal Brisbane and Women's Hospital, ak University of Queensland, Brisbane, Queensland, Ostrali
12Enstiti pou ematoloji eksperimantal ak medikaman transfizyon ak Sant pou Maladi Ra, Universitätsklinikum Bonn, Bonn, Nordrhein-Westfalen, Almay
13Hemocentro UNICAMP, Depatman Medsin Entèn, Lekòl Syans Medikal, Inivèsite Campinas, Campinas, Sao Paulo, Brezil
14Fondasyon IRCCS Ca' Granda - Ospedale Maggiore Policlinico, UOC Medicina Generale, Angelo Bianchi Bonomi Sant emofili ak tronboz ak Fondazione Luigi Villa, ak Depatman Fizyoloji ak Transplantasyon, Università degli Studi di Milano, Milan, Lombardi, Itali.
15Sant Tretman Òtopedik Emofili, Los Angeles, Kalifòni, Etazini
16Sant pou senyen ak twoub koagulasyon, University of Minnesota, Minneapolis, Minnesota, Etazini
17Oxford University Hospitals National Health Service Foundation Trust, University of Oxford, ak Oxford National Institute for Health Research Biomedical Research Centre, Oxford, Angletè, Wayòm Ini.
18BioMarin Pharmaceutical, Inc., Novato, Kalifòni, Etazini

19Depatman Pedyatri ak Patoloji, University of Michigan, Ann Arbor, Michigan, Etazini

Kle Done Pwen

Valoctocogene Roxaparvovec Rezime Sekirite pou Ane 2
Anualize Pousantaj Senyen trete/FVIII Itilizasyon

Rezilta faz 3 GENER8-1 etid la sou pasyan ki gen emofili A ki te trete ak valoctocogene roxaparvovec montre yon rediksyon konsistan nan senyen trete/ane ak itilizasyon anyèl FVIII soti nan ane 1 rive nan ane 2 konpare ak debaz. Valè debaz yo te soti nan peryòd koleksyon done potansyèl 6 mwa nan etid noninterventional BMN 270-902.

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