Høydepunkter fra den 14. årlige kongressen til EAHAD
Etranacogene Dezaparvovec (AAV5-Padua hFIX-variant), en forbedret vektor for genoverføring hos voksne med alvorlig eller moderat-alvorlig hemofili B: To års data fra en fase 2b-prøve
Annette von Drygalski, MD, PharmD1, Adam Giermasz, MD, PhD2, Giancarlo Castaman, MD3, Nigel S. Key, MBChB4, Susan U. Lattimore, RN5, Frank WG Leebeek, MD6, Wolfgang A. Miesbach, MD, PhD7, Michael Recht, MD, PhD5, Esteban Gomez, MD8, Robert Gut, MD, PhD9og Steven W. Pipe, MD10
1University of California San Diego, La Jolla, CA, USA
2University of California Davis, Sacramento, CA, USA
3Azienda Ospedaliera Universitaria Careggi, Firenze, Italia
4University of North Carolina, Chapel Hill, USA
5Oregon Health and Science University, Portland, USA
6Erasmus University Medical Center, Rotterdam, Nederland
7Universitetssykehuset Frankfurt, Tyskland
8Phoenix Children's Hospital, Phoenix, USA
9uniQure Inc, Lexington, MA, USA
10University of Michigan, Ann Arbor, MI, USA
Annette von Drygalski, MD, PharmD1, Adam Giermasz, MD, PhD2, Giancarlo Castaman, MD3, Nigel S. Key, MBChB4, Susan U. Lattimore, RN5, Frank WG Leebeek, MD6, Wolfgang A. Miesbach, MD, PhD7, Michael Recht, MD, PhD5, Esteban Gomez, MD8, Robert Gut, MD, PhD9og Steven W. Pipe, MD10
1University of California San Diego, La Jolla, CA, USA
2University of California Davis, Sacramento, CA, USA
3Azienda Ospedaliera Universitaria Careggi, Firenze, Italia
4University of North Carolina, Chapel Hill, USA
5Oregon Health and Science University, Portland, USA
6Erasmus University Medical Center, Rotterdam, Nederland
7Universitetssykehuset Frankfurt, Tyskland
8Phoenix Children's Hospital, Phoenix, USA
9uniQure Inc, Lexington, MA, USA
10University of Michigan, Ann Arbor, MI, USA