Høydepunkter fra den 14. årlige kongressen til EAHAD
Effektivitet og sikkerhet av Etranacogene Dezaparvovec hos voksne med alvorlig eller moderat alvorlig hemofili B: Første data fra fase 3-håp-B gentheraprøve
Steven W Pipe, MD1, Michael Recht, MD, PhD2, Nigel S Key, MBChB3, Frank WG Leebeek, MD4, Giancarlo Castaman, MD5, Susan U Lattimore, RN6, Paul van der Valk, MD6, Kathelijne Peerlinck, MD, PhD7, Michiel Coppens, MD, PhD8, Niamh O'Connell, MB PhD9, K. John Pasi, MBChB, PhD10, Peter Kampmann, MD11, Karina Meijer, MD, PhD.12, Annette von Drygalski, MD, PharmD13, Guy Young, MD14, Cedric Hermans, MD15, Jan Astermark, MD, PhD16, Robert Klamroth, MD, PhD17, Richard S Lemons, MD, PhD18, Nathan Visweshwar, MD19, Shelley Crary, MD20, Rashid Kazmi, MBBS21, Emily Symington, MBChB22, Miguel A Escobar, MD23, Esteban Gomez, MD24, Rebecca Kruse-Jarres, MD, MPH25, Adam Kotowski, MD26, Doris Quon, MD, PhD27, Michael Wang, MD28, Allison P Wheeler, MD29, Eileen K Sawyer, PhD30, Stephanie Verweij30, Valerie Colletta, MSc30, Naghmana Bajwa, MD, Robert Gut, MD, PD30, Wolfgang Miesbach, MD, PhD31
1University of Michigan, Ann Arbor, MI, USA
2Oregon Health & Science University, Portland, OR, USA
3University of North Carolina, Chapel Hill, NC, USA
4Erasmus MC, University Medical Center, Rotterdam, Nederland
5Center for Bleeding Disorders and coagulation, Careggi University Hospital, Firenze, Italia
6Universitair Medisch Centrum Utrecht, Nederland
7Institutt for vaskulær medisin og hemostase og hemofili senter, universitetssykehus Leuven, Leuven, Belgia
8Institutt for vaskulær medisin, Amsterdam kardiovaskulære vitenskaper, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Nederland
9The National Coagulation Centre, St James's Hospital, Dublin, Irland
10Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, Storbritannia
11Rigshospitalet, København, Danmark
12Universitetsmedisinske senter Groningen, Groningen, Nederland
13University of California San Diego, La Jolla, CA, USA
14Barnesykehus Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA
15Cliniques Universitaires Saint ‐ Luc, Université Catholique de Louvain, Brussel, Belgia
16Skåne universitetssykehus, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, Tyskland
18University of Utah, Salt Lake City, UT, USA
19University of South Florida, Tampa, FL, USA
20University of Arkansas for medisinske vitenskaper, AR, USA
21Universitetssykehus Southampton NHS Foundation Trust; Southampton, Storbritannia
22Cambridge University - Addenbrooke's Hospital, Cambridge, Storbritannia
23University of Texas Health Science Center i Houston, Houston, TX, USA
24Phoenix Children's Hospital, Phoenix USA
25Bloodworks Northwest, Seattle, WA
26WNY BloodCare, NY, USA
27Los Angeles Ortopedic Hospital, Orthopedic Hemophilia Treatment Center, Los Angeles, CA, USA
28University of Colorado Hemophilia and Thrombosis Center, Anschutz Medical Campus, Aurora, CO
29Vanderbilt University Medical Center, Nashville, TN, USA
30uniQure BV, Amsterdam, Nederland / uniQure Inc. Lexington, MA, USA
31Universitetssykehuset Frankfurt, Frankfurt, Tyskland
Steven W Pipe, MD1, Michael Recht, MD, PhD2, Nigel S Key, MBChB3, Frank WG Leebeek, MD4, Giancarlo Castaman, MD5, Susan U Lattimore, RN6, Paul van der Valk, MD6, Kathelijne Peerlinck, MD, PhD7, Michiel Coppens, MD, PhD8, Niamh O'Connell, MB PhD9, K. John Pasi, MBChB, PhD10, Peter Kampmann, MD11, Karina Meijer, MD, PhD.12, Annette von Drygalski, MD, PharmD13, Guy Young, MD14, Cedric Hermans, MD15, Jan Astermark, MD, PhD16, Robert Klamroth, MD, PhD17, Richard S Lemons, MD, PhD18, Nathan Visweshwar, MD19, Shelley Crary, MD20, Rashid Kazmi, MBBS21, Emily Symington, MBChB22, Miguel A Escobar, MD23, Esteban Gomez, MD24, Rebecca Kruse-Jarres, MD, MPH25, Adam Kotowski, MD26, Doris Quon, MD, PhD27, Michael Wang, MD28, Allison P Wheeler, MD29, Eileen K Sawyer, PhD30, Stephanie Verweij30, Valerie Colletta, MSc30, Naghmana Bajwa, MD, Robert Gut, MD, PD30, Wolfgang Miesbach, MD, PhD31
1University of Michigan, Ann Arbor, MI, USA
2Oregon Health & Science University, Portland, OR, USA
3University of North Carolina, Chapel Hill, NC, USA
4Erasmus MC, University Medical Center, Rotterdam, Nederland
5Center for Bleeding Disorders and coagulation, Careggi University Hospital, Firenze, Italia
6Universitair Medisch Centrum Utrecht, Nederland
7Institutt for vaskulær medisin og hemostase og hemofili senter, universitetssykehus Leuven, Leuven, Belgia
8Institutt for vaskulær medisin, Amsterdam kardiovaskulære vitenskaper, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Nederland
9The National Coagulation Centre, St James's Hospital, Dublin, Irland
10Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, Storbritannia
11Rigshospitalet, København, Danmark
12Universitetsmedisinske senter Groningen, Groningen, Nederland
13University of California San Diego, La Jolla, CA, USA
14Barnesykehus Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA
15Cliniques Universitaires Saint ‐ Luc, Université Catholique de Louvain, Brussel, Belgia
16Skåne universitetssykehus, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, Tyskland
18University of Utah, Salt Lake City, UT, USA
19University of South Florida, Tampa, FL, USA
20University of Arkansas for medisinske vitenskaper, AR, USA
21Universitetssykehus Southampton NHS Foundation Trust; Southampton, Storbritannia
22Cambridge University - Addenbrooke's Hospital, Cambridge, Storbritannia
23University of Texas Health Science Center i Houston, Houston, TX, USA
24Phoenix Children's Hospital, Phoenix USA
25Bloodworks Northwest, Seattle, WA
26WNY BloodCare, NY, USA
27Los Angeles Ortopedic Hospital, Orthopedic Hemophilia Treatment Center, Los Angeles, CA, USA
28University of Colorado Hemophilia and Thrombosis Center, Anschutz Medical Campus, Aurora, CO
29Vanderbilt University Medical Center, Nashville, TN, USA
30uniQure BV, Amsterdam, Nederland / uniQure Inc. Lexington, MA, USA
31Universitetssykehuset Frankfurt, Frankfurt, Tyskland