Pwen esansyèl nan ISTH 2021 Kongrè a
Efikasite ak sekirite nan Valoctocogene Roxaparvovec Adeno-asosye viris transfè jèn pou emofili grav A: Rezilta ki soti nan faz 3 GENEr8-1 jijman an
MC Ozelo1, J. Mahlangu2, KJ. Pasi3, A. Giermasz4, AD Leavitt5, M. Laffan6, E. Symington7, DV Quon8, J.-D. Wang9, K. Peerlinck11, S. Tiyo11, B. Madan12, NS kle13, GF Pierce14, B. O'Mahony15,16, R. Kaczmarek17,18, A. Lawal19, M. Huang19, WY Wong19, B. Kim19, GENEr8-1 Gwoup esè
1Hemocentro UNICAMP, Depatman Medsin Entèn, Lekòl Syans Medikal, University of Campinas, Campinas, Brezil
2Emophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Hospital Hospital, University of the Witwatersrand and NHLS, Johannesburg, Lafrik di sid
3Barts ak London Medsin Lekòl la ak Dexter, London, Wayòm Ini
4Emofili Sant Tretman, University of California Davis, Sacramento, Etazini
5Inivèsite Kalifòni, San Francisco, Etazini
6Sant pou ematoloji, Imperial College London, London, Wayòm Ini
7Cambridge Inivèsite Lopital NHS Fondasyon Trust, Cambridge, Wayòm Ini
8Orthopedic Emophilia Treatment Center, Los Angeles, Etazini
9Sant pou Maladi Ra ak Emofili, Taichung Veteran Lopital Jeneral, Taichung, Taiwan, Pwovens nan Lachin
10Depatman Medsin vaskilè ak Ememostazi ak Emofili Sant, Inivèsite Lopital Louvain, Louvain, Bèljik
11Depatman Pedyatri ak Patoloji, University of Michigan, Ann Arbor, Etazini
12Fondasyon NHS Guy's & St. Thomas ', London, Wayòm Ini
13UNC San Rechèch Sant, University of North Carolina, Chapel Hill, Etazini
14Konsiltan Endepandan, La Jolla, Etazini
15Irish Haemophilia Society, Dublin, Iland
16Trinity College, Dublin, Iland
17Depatman Pedyatri, Indiana Inivèsite Lekòl Medsin, IUPUI-Wells Sant pou Rechèch Pedyat, Indianapolis, Etazini
18Laboratwa nan glikobioloji, Hirszfeld Enstiti pou iminoloji ak terapi eksperimantal, Wroclaw, Polòy
19BioMarin famasetik Inc, Novato, Etazini
MC Ozelo1, J. Mahlangu2, KJ. Pasi3, A. Giermasz4, AD Leavitt5, M. Laffan6, E. Symington7, DV Quon8, J.-D. Wang9, K. Peerlinck11, S. Tiyo11, B. Madan12, NS kle13, GF Pierce14, B. O'Mahony15,16, R. Kaczmarek17,18, A. Lawal19, M. Huang19, WY Wong19, B. Kim19, GENEr8-1 Gwoup esè
1Hemocentro UNICAMP, Depatman Medsin Entèn, Lekòl Syans Medikal, University of Campinas, Campinas, Brezil
2Emophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Hospital Hospital, University of the Witwatersrand and NHLS, Johannesburg, Lafrik di sid
3Barts ak London Medsin Lekòl la ak Dexter, London, Wayòm Ini
4Emofili Sant Tretman, University of California Davis, Sacramento, Etazini
5Inivèsite Kalifòni, San Francisco, Etazini
6Sant pou ematoloji, Imperial College London, London, Wayòm Ini
7Cambridge Inivèsite Lopital NHS Fondasyon Trust, Cambridge, Wayòm Ini
8Orthopedic Emophilia Treatment Center, Los Angeles, Etazini
9Sant pou Maladi Ra ak Emofili, Taichung Veteran Lopital Jeneral, Taichung, Taiwan, Pwovens nan Lachin
10Depatman Medsin vaskilè ak Ememostazi ak Emofili Sant, Inivèsite Lopital Louvain, Louvain, Bèljik
11Depatman Pedyatri ak Patoloji, University of Michigan, Ann Arbor, Etazini
12Fondasyon NHS Guy's & St. Thomas ', London, Wayòm Ini
13UNC San Rechèch Sant, University of North Carolina, Chapel Hill, Etazini
14Konsiltan Endepandan, La Jolla, Etazini
15Irish Haemophilia Society, Dublin, Iland
16Trinity College, Dublin, Iland
17Depatman Pedyatri, Indiana Inivèsite Lekòl Medsin, IUPUI-Wells Sant pou Rechèch Pedyat, Indianapolis, Etazini
18Laboratwa nan glikobioloji, Hirszfeld Enstiti pou iminoloji ak terapi eksperimantal, Wroclaw, Polòy
19BioMarin famasetik Inc, Novato, Etazini