Hápunktar frá sýndarráðstefnunni WFH 2020
Fyrstu manna fjögurra ára eftirfylgni rannsókn á varanlegri meðferðarvirkni og öryggi: AAV genameðferð með Valoctocogene Roxaparvovec fyrir alvarlegri dreyrasýki 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, læknir, FRCP, FRCPath6; Michael Laffan, DM, FRCP, FRCPath7; Chris B. Russell, doktorsgráðu3; Mingjin Li, MSc3; Benjamin Kim, læknir, MPhil3; Glenn F. Pierce, læknir, doktor8; Wing Yen Wong, læknir3
1Barts og London School of Medicine and Dentistry, London, UK
2Háskólasjúkrahús Southampton, Southampton, Bretlandi
3BioMarin Pharmaceutical Inc., Novato, CA, Bandaríkjunum
4Háskólasjúkrahús Birmingham NHS Foundation Trust, Birmingham, Bretlandi
5Cambridge háskólasjúkrahús NHS Foundation Trust, Cambridge, Bretlandi
6NHS Foundation Trust Guy's & St. Thomas, London, Bretlandi
7Centre for Hematology, Imperial College London, London, UK.
8Ráðgjafi, La Jolla, CA, Bandaríkjunum
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, læknir, FRCP, FRCPath6; Michael Laffan, DM, FRCP, FRCPath7; Chris B. Russell, doktorsgráðu3; Mingjin Li, MSc3; Benjamin Kim, læknir, MPhil3; Glenn F. Pierce, læknir, doktor8; Wing Yen Wong, læknir3
1Barts og London School of Medicine and Dentistry, London, UK
2Háskólasjúkrahús Southampton, Southampton, Bretlandi
3BioMarin Pharmaceutical Inc., Novato, CA, Bandaríkjunum
4Háskólasjúkrahús Birmingham NHS Foundation Trust, Birmingham, Bretlandi
5Cambridge háskólasjúkrahús NHS Foundation Trust, Cambridge, Bretlandi
6NHS Foundation Trust Guy's & St. Thomas, London, Bretlandi
7Centre for Hematology, Imperial College London, London, UK.
8Ráðgjafi, La Jolla, CA, Bandaríkjunum