Punti salienti del vertice virtuale WFH 2020
Studio di follow-up di quattro anni sull'uomo per la prima volta sull'efficacia terapeutica e sulla sicurezza durevoli: terapia genica AAV con valoctocogene Roxaparvovec per emofilia grave A
K. John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH1; Savita Rangarajan, MBBS, FRCP, FRCPath2; Nina Mitchell, MB, BCir3; Will Lester, MB, ChB, PhD, FRCP, FRCPath4; Emily Symington, laurea, MBBS, MRCP, FRCPath5; Bella Madan, MD, FRCP, FRCPath6; Michael Laffan, DM, FRCP, FRCPath7; Chris B. Russell, PhD3; Mingjin Li, MSc3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, PhD8; Wing Yen Wong, dottore in medicina3
1Barts e la London School of Medicine and Dentistry, Londra, Regno Unito
2University Hospital Southampton, Southampton, Regno Unito
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4Ospedali universitari Birmingham NHS Foundation Trust, Birmingham, Regno Unito
5Ospedali dell'Università di Cambridge NHS Foundation Trust, Cambridge, Regno Unito
6Guy's & St. Thomas 'NHS Foundation Trust, Londra, Regno Unito
7Centre for Hematology, Imperial College London, Londra, Regno Unito.
8Consulente, La Jolla, California, USA
K. John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH1; Savita Rangarajan, MBBS, FRCP, FRCPath2; Nina Mitchell, MB, BCir3; Will Lester, MB, ChB, PhD, FRCP, FRCPath4; Emily Symington, laurea, MBBS, MRCP, FRCPath5; Bella Madan, MD, FRCP, FRCPath6; Michael Laffan, DM, FRCP, FRCPath7; Chris B. Russell, PhD3; Mingjin Li, MSc3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, PhD8; Wing Yen Wong, dottore in medicina3
1Barts e la London School of Medicine and Dentistry, Londra, Regno Unito
2University Hospital Southampton, Southampton, Regno Unito
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4Ospedali universitari Birmingham NHS Foundation Trust, Birmingham, Regno Unito
5Ospedali dell'Università di Cambridge NHS Foundation Trust, Cambridge, Regno Unito
6Guy's & St. Thomas 'NHS Foundation Trust, Londra, Regno Unito
7Centre for Hematology, Imperial College London, Londra, Regno Unito.
8Consulente, La Jolla, California, USA