Hlavné body z virtuálneho samitu WFH 2020
Prvé štvorročné štúdium trvajúcej terapeutickej účinnosti a bezpečnosti u ľudí: Génová terapia AAV valoktogénom Roxaparvovec pre závažnú hemofíliu 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, PhD3; Mingjin Li, MSc3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, PhD8; MUDr. Wing Yen Wong3
1Barts a London School of Medicine and Dentistry, London, UK
2University Hospital Southampton, Southampton, Spojené kráľovstvo
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4University Hospital, Birmingham, NHS Foundation Trust, Birmingham, Spojené kráľovstvo
5NHS Foundation Trust, Cambridge University Hospital, Cambridge, UK
6Guy's & St. Thomas 'NHS Foundation Trust, Londýn, Spojené kráľovstvo
7Centrum pre hematológiu, Imperial College London, Londýn, Spojené kráľovstvo.
8Konzultant, La Jolla, CA, USA
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, PhD3; Mingjin Li, MSc3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, PhD8; MUDr. Wing Yen Wong3
1Barts a London School of Medicine and Dentistry, London, UK
2University Hospital Southampton, Southampton, Spojené kráľovstvo
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4University Hospital, Birmingham, NHS Foundation Trust, Birmingham, Spojené kráľovstvo
5NHS Foundation Trust, Cambridge University Hospital, Cambridge, UK
6Guy's & St. Thomas 'NHS Foundation Trust, Londýn, Spojené kráľovstvo
7Centrum pre hematológiu, Imperial College London, Londýn, Spojené kráľovstvo.
8Konzultant, La Jolla, CA, USA