Destaquem de la Cimera Virtual WFH 2020
Un primer estudi de quatre anys de seguiment humà de l'eficàcia i la seguretat terapèutiques duradores: la gènera teràpia AAV amb valoctocogen Roxaparvovec per a una hemofília greu A
K. John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH1; Savita Rangarajan, MBBS, FRCP, FRCPath2; Nina Mitchell, MB, BChir3; Will Lester, MB, ChB, PhD, FRCP, FRCPath4; Emily Symington, BSc, MBBS, MRCP, FRCPath5; Bella Madan, MD, FRCP, FRCPath6; Michael Laffan, DM, FRCP, FRCPath7; Chris B. Russell, doctor3; Mingjin Li, MSc3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, doctor8; Wing Yen Wong, MD3
1Barts i la London School of Medicine and Odontologia, Londres, Regne Unit
2Hospital Universitari de Southampton, Southampton, Regne Unit
3BioMarin Pharmaceutical Inc., Novato, Califòrnia, EUA
4Hospitals universitaris Birmingham NHS Foundation Trust, Birmingham, Regne Unit
5Hospitals de la Universitat de Cambridge NHS Foundation Trust, Cambridge, Regne Unit
6Guy's & St. Thomas 'NHS Foundation Trust, Londres, Regne Unit
7Centre d’Hematologia, Imperial College London, Londres, Regne Unit.
8Consultora, La Jolla, CA, EUA
K. John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH1; Savita Rangarajan, MBBS, FRCP, FRCPath2; Nina Mitchell, MB, BChir3; Will Lester, MB, ChB, PhD, FRCP, FRCPath4; Emily Symington, BSc, MBBS, MRCP, FRCPath5; Bella Madan, MD, FRCP, FRCPath6; Michael Laffan, DM, FRCP, FRCPath7; Chris B. Russell, doctor3; Mingjin Li, MSc3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, doctor8; Wing Yen Wong, MD3
1Barts i la London School of Medicine and Odontologia, Londres, Regne Unit
2Hospital Universitari de Southampton, Southampton, Regne Unit
3BioMarin Pharmaceutical Inc., Novato, Califòrnia, EUA
4Hospitals universitaris Birmingham NHS Foundation Trust, Birmingham, Regne Unit
5Hospitals de la Universitat de Cambridge NHS Foundation Trust, Cambridge, Regne Unit
6Guy's & St. Thomas 'NHS Foundation Trust, Londres, Regne Unit
7Centre d’Hematologia, Imperial College London, Londres, Regne Unit.
8Consultora, La Jolla, CA, EUA