Høydepunkter fra WFH Virtual Summit 2020
Først-i-menneske Fire-årig oppfølgingsstudie av holdbar terapeutisk effektivitet og sikkerhet: AAV genterapi med Valoctocogen Roxaparvovec for alvorlig hemofili 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, doktorgrad3; Mingjin Li, MSc3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, PhD8; Wing Yen Wong, MD3
1Barts og London School of Medicine and Dentistry, London, UK
2University Hospital Southampton, Southampton, Storbritannia
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4Universitetssykehus Birmingham NHS Foundation Trust, Birmingham, Storbritannia
5Cambridge University Hospitales NHS Foundation Trust, Cambridge, Storbritannia
6Guy's & St. Thomas 'NHS Foundation Trust, London, Storbritannia
7Centre for Hematology, Imperial College London, London, Storbritannia.
8Konsulent, 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, doktorgrad3; Mingjin Li, MSc3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, PhD8; Wing Yen Wong, MD3
1Barts og London School of Medicine and Dentistry, London, UK
2University Hospital Southampton, Southampton, Storbritannia
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4Universitetssykehus Birmingham NHS Foundation Trust, Birmingham, Storbritannia
5Cambridge University Hospitales NHS Foundation Trust, Cambridge, Storbritannia
6Guy's & St. Thomas 'NHS Foundation Trust, London, Storbritannia
7Centre for Hematology, Imperial College London, London, Storbritannia.
8Konsulent, La Jolla, CA, USA