Højdepunkter fra WFH Virtual Summit 2020
Første-i-menneske Fire-årig opfølgningsundersøgelse af holdbar terapeutisk effektivitet og sikkerhed: AAV-genterapi med Valoctocogen Roxaparvovec for alvorlig hæmofili 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, ph.d.3; Mingjin Li, cand3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, ph.d.8; Wing Yen Wong, MD3
1Barts og London School of Medicine and Dentistry, London, UK
2Universitetshospital Southampton, Southampton, Storbritannien
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4Universitetshospitaler Birmingham NHS Foundation Trust, Birmingham, UK
5Cambridge University Hospitaler NHS Foundation Trust, Cambridge, UK
6Guy's & St. Thomas 'NHS Foundation Trust, London, UK
7Center for Hematology, Imperial College London, London, UK.
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, ph.d.3; Mingjin Li, cand3; Benjamin Kim, MD, MPhil3; Glenn F. Pierce, MD, ph.d.8; Wing Yen Wong, MD3
1Barts og London School of Medicine and Dentistry, London, UK
2Universitetshospital Southampton, Southampton, Storbritannien
3BioMarin Pharmaceutical Inc., Novato, CA, USA
4Universitetshospitaler Birmingham NHS Foundation Trust, Birmingham, UK
5Cambridge University Hospitaler NHS Foundation Trust, Cambridge, UK
6Guy's & St. Thomas 'NHS Foundation Trust, London, UK
7Center for Hematology, Imperial College London, London, UK.
8Konsulent, La Jolla, CA, USA