Pwen esansyèl ki soti nan reyinyon 62th ASH Anyèl ak Ekspozisyon an
Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), yon vektè amelyore pou transfè jèn nan granmoun ki gen emofil grav oswa modere-grav: de ane done ki sòti nan yon faz 2b esè.
Annette von Drygalski, MD, Pharm D.1, Adan Giermasz, MD, PhD2, Giancarlo Castaman, MD3, Nigel S. Key, MD4,5, Susan U. Lattimore, RN6, Frank WG Leebeek, MD, PhD7, Wolfgang A. Miesbach, MD8, Michael Recht, MD, PhD9, Esteban Gomez, MD10, Robert Gut, MD, PhD11, ak Steven W. Pipe, MD12
1Depatman Medsin, Divizyon ematoloji / nkoloji, UC San Diego Sante, San Diego, CA
2Inivèsite Kalifòni Davis, Sacramento, CA
3Azienda Ospedaliera Università Careggi, Florence, Itali
4Inivèsite North Carolina nan Chapel Hill, Chapel Hill, NC
5Divizyon ematoloji, University of North Carolina nan Chapel Hill, Chapel Hill, NC
6Oregon Sante ak Syans Inivèsite, Portland, OSWA
7Sant Medikal Inivèsite Erasmus, Rotterdam, Netherlands
8Emofili Sant, Inivèsite Lopital Frankfurt, Frankfurt, Almay
9Oregon Sante ak Syans Inivèsite, Portland, OSWA
10Lopital Timoun Phoenix, Phoenix, AZ
11uniQure Inc, Lexington, MA
12University of Michigan, Ann Arbor, MI