Utrinki z virtualnega vrha WFH 2020
Prvoletna študija človeka o trajni terapevtski učinkovitosti in varnosti: genska terapija AAV z valoktokogenom Roxaparvovec za hudo hemofilijo 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, univ.dipl.inž., MBBS, MRCP, FRCPath5; Bella Madan, dr. Med., FRCP, FRCPath6; Michael Laffan, DM, FRCP, FRCPath7; Chris B. Russell, dr3; Mingjin Li, mag3; Benjamin Kim, dr. Med., MPhil3; Glenn F. Pierce, dr. Med8; Wing Yen Wong, dr. Med3
1Barts in London School of Medicine and Dentistry, London, Velika Britanija
2University Hospital Southampton, Southampton, Združeno kraljestvo
3BioMarin Pharmaceutical Inc., Novato, Kalifornija, ZDA
4Univerzitetne bolnišnice Birmingham NHS Foundation Trust, Birmingham, Velika Britanija
5Svetovalne bolnišnice Cambridge NHS Foundation Trust, Cambridge, Združeno kraljestvo
6Guy's & St. Thomas 'NHS Foundation Trust, London, Velika Britanija
7Center za hematologijo, Imperial College London, London, Velika Britanija.
8Svetovalec, La Jolla, Kalifornija, ZDA
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, univ.dipl.inž., MBBS, MRCP, FRCPath5; Bella Madan, dr. Med., FRCP, FRCPath6; Michael Laffan, DM, FRCP, FRCPath7; Chris B. Russell, dr3; Mingjin Li, mag3; Benjamin Kim, dr. Med., MPhil3; Glenn F. Pierce, dr. Med8; Wing Yen Wong, dr. Med3
1Barts in London School of Medicine and Dentistry, London, Velika Britanija
2University Hospital Southampton, Southampton, Združeno kraljestvo
3BioMarin Pharmaceutical Inc., Novato, Kalifornija, ZDA
4Univerzitetne bolnišnice Birmingham NHS Foundation Trust, Birmingham, Velika Britanija
5Svetovalne bolnišnice Cambridge NHS Foundation Trust, Cambridge, Združeno kraljestvo
6Guy's & St. Thomas 'NHS Foundation Trust, London, Velika Britanija
7Center za hematologijo, Imperial College London, London, Velika Britanija.
8Svetovalec, La Jolla, Kalifornija, ZDA