Mga highlight mula sa WFH Virtual Summit 2020
First-in-human Four-year Follow-up Study of Durable Therapeutic Efficacy and Safety: AAV Gene Therapy With Valoctocogene Roxaparvovec para sa Severe Haemophilia A
K. John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH1; Savita Rangarajan, MBBS, FRCP, FRCPath2; Nina Mitchell, MB, BChir3; Ang 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; Wing Yen Wong, MD3
1Barts at ang London School of Medicine at Dentistry, London, UK
2University Hospital Southampton, Southampton, UK
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4Mga Ospital ng Unibersidad ng Birmingham NHS Foundation Trust, Birmingham, UK
5Mga Ospital ng Cambridge University NHS Foundation Trust, Cambridge, UK
6Guy & St Thomas 'NHS Foundation Trust, London, UK
7Center para sa Hematology, Imperial College London, London, UK.
8Konsulta, La Jolla, CA, USA
K. John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH1; Savita Rangarajan, MBBS, FRCP, FRCPath2; Nina Mitchell, MB, BChir3; Ang 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; Wing Yen Wong, MD3
1Barts at ang London School of Medicine at Dentistry, London, UK
2University Hospital Southampton, Southampton, UK
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4Mga Ospital ng Unibersidad ng Birmingham NHS Foundation Trust, Birmingham, UK
5Mga Ospital ng Cambridge University NHS Foundation Trust, Cambridge, UK
6Guy & St Thomas 'NHS Foundation Trust, London, UK
7Center para sa Hematology, Imperial College London, London, UK.
8Konsulta, La Jolla, CA, USA