Mga Highlight Mula sa Ika-62 na Taunang ASH ng Pagpupulong at Paglalahad
Etranacogene Dezaparvovec (variant AAV5-Padua hFIX), isang Pinahusay na Vector para sa Paglipat ng Gene sa Mga Matanda na May Malubha o Katamtamang-Malubhang Hemophilia B: Dalawang Taon na Data Mula sa isang Phase 2b Trial
Annette von Drygalski, MD, Pharm D1, Adam 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, at Steven W. Pipe, MD12
1Kagawaran ng Medisina, Division of Hematology / Oncology, UC San Diego Health, San Diego, CA
2Unibersidad ng California Davis, Sacramento, CA
3Azienda Ospedaliera Universitaria Careggi, Florence, Italya
4University of North Carolina sa Chapel Hill, Chapel Hill, NC
5Dibisyon ng Hematology, University of North Carolina sa Chapel Hill, Chapel Hill, NC
6Oregon Health & Science University, Portland, OR
7Erasmus University Medical Center, Rotterdam, Netherlands
8Hemophilia Center, University Hospital Frankfurt, Frankfurt, Germany
9Oregon Health and Science University, Portland, OR
10Phoenix Children's Hospital, Phoenix, AZ
11uniQure Inc, Lexington, MA
12University of Michigan, Ann Arbor, MI