Punti Ewlenin Mit-62 Laqgħa u Espożizzjoni Annwali tal-ASH
Etranacogene Dezaparvovec (varjant AAV5-Padova hFIX), Vettur Imtejjeb għat-Trasferiment tal-Ġeni f'Adulti b'Hemofilja B Severa jew Moderata-Severa: Dejta ta 'sentejn minn Prova ta' Fażi 2b
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, u Steven W. Pipe, MD12
1Dipartiment tal-Mediċina, Diviżjoni tal-Ematoloġija / Onkoloġija, UC San Diego Health, San Diego, CA
2Università ta 'California Davis, Sacramento, CA
3Azienda Ospedaliera Universitaria Careggi, Firenze, l-Italja
4Università ta 'North Carolina f'Chapel Hill, Chapel Hill, NC
5Diviżjoni tal-Ematoloġija, Università ta 'North Carolina f'Chapel Hill, Chapel Hill, NC
6Oregon Health & Science University, Portland, OR
7Ċentru Mediku tal-Università Erasmus, Rotterdam, l-Olanda
8Ċentru tal-Emofilja, Sptar Universitarju Frankfurt, Frankfurt, il-Ġermanja
9Oregon Health and Science University, Portland, OR
10Isptar tat-Tfal ta 'Phoenix, Phoenix, AZ
11uniQure Inc, Lexington, MA
12Università ta 'Michigan, Ann Arbor, MI