Højdepunkter fra ISTH 2021 -kongressen
Effekt og sikkerhed af Valoctocogen Roxaparvovec Adeno-associeret virusgenoverførsel til alvorlig hæmofili A: Resultater fra fase 3 GENEr8-1-forsøget
MC Ozelo1, J. Mahlangu2, KJ. Pasi3, A. Giermasz4, AD Leavitt5, M. Laffan6, E. Symington7, DV Quon8, J.-D. Wang9, K. Peerlinck11, S. Pipe11, B. Madan12, NS -nøgle13, GF Pierce14, B. O'Mahony15,16, R. Kaczmarek17,18, A. Lawal19, M. Huang19, WY Wong19, B. Kim19, GENEr8-1 prøvegruppe
1Hemocentro UNICAMP, Institut for Intern Medicin, Institut for Medicinsk Videnskab, University of Campinas, Campinas, Brasilien
2Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand og NHLS, Johannesburg, Sydafrika
3Barts og London School of Medicine and Dentistry, London, Storbritannien
4Hemophilia Treatment Center, University of California Davis, Sacramento, USA
5University of California, San Francisco, USA
6Center for Hematology, Imperial College London, London, Storbritannien
7Cambridge University Hospitals NHS Foundation Trust, Cambridge, Storbritannien
8Orthopedic Hemophilia Treatment Center, Los Angeles, USA
9Center for sjælden sygdom og hæmofili, Taichung Veterans General Hospital, Taichung, Taiwan, Kina
10Institut for Vaskulær Medicin og Hæmostase og Hæmofili Center, Universitetshospitaler Leuven, Leuven, Belgien
11Department of Pediatrics and Pathology, University of Michigan, Ann Arbor, USA
12Guy's & St. Thomas 'NHS Foundation Trust, London, Storbritannien
13UNC Blood Research Center, University of North Carolina, Chapel Hill, USA
14Uafhængig konsulent, La Jolla, USA
15Irish Haemophilia Society, Dublin, Irland
16Trinity College, Dublin, Irland
17Department of Pediatrics, Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, Indianapolis, USA
18Laboratorium for glykobiologi, Hirszfeld Institute of Immunology and Experimental Therapy, Wroclaw, Polen
19BioMarin Pharmaceutical Inc., Novato, USA
MC Ozelo1, J. Mahlangu2, KJ. Pasi3, A. Giermasz4, AD Leavitt5, M. Laffan6, E. Symington7, DV Quon8, J.-D. Wang9, K. Peerlinck11, S. Pipe11, B. Madan12, NS -nøgle13, GF Pierce14, B. O'Mahony15,16, R. Kaczmarek17,18, A. Lawal19, M. Huang19, WY Wong19, B. Kim19, GENEr8-1 prøvegruppe
1Hemocentro UNICAMP, Institut for Intern Medicin, Institut for Medicinsk Videnskab, University of Campinas, Campinas, Brasilien
2Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand og NHLS, Johannesburg, Sydafrika
3Barts og London School of Medicine and Dentistry, London, Storbritannien
4Hemophilia Treatment Center, University of California Davis, Sacramento, USA
5University of California, San Francisco, USA
6Center for Hematology, Imperial College London, London, Storbritannien
7Cambridge University Hospitals NHS Foundation Trust, Cambridge, Storbritannien
8Orthopedic Hemophilia Treatment Center, Los Angeles, USA
9Center for sjælden sygdom og hæmofili, Taichung Veterans General Hospital, Taichung, Taiwan, Kina
10Institut for Vaskulær Medicin og Hæmostase og Hæmofili Center, Universitetshospitaler Leuven, Leuven, Belgien
11Department of Pediatrics and Pathology, University of Michigan, Ann Arbor, USA
12Guy's & St. Thomas 'NHS Foundation Trust, London, Storbritannien
13UNC Blood Research Center, University of North Carolina, Chapel Hill, USA
14Uafhængig konsulent, La Jolla, USA
15Irish Haemophilia Society, Dublin, Irland
16Trinity College, Dublin, Irland
17Department of Pediatrics, Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, Indianapolis, USA
18Laboratorium for glykobiologi, Hirszfeld Institute of Immunology and Experimental Therapy, Wroclaw, Polen
19BioMarin Pharmaceutical Inc., Novato, USA