Highlights From the 14th Annual Congress of EAHAD
Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: First Data From the Phase 3 Hope-B Gene Therapy Trial
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, M.D., 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, P.D30, 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, The Netherlands
5Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy
6Universitair Medisch Centrum Utrecht, Netherlands
7Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium
8Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands
9The National Coagulation Centre, St James’s Hospital, Dublin, Ireland
10Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, UK
11Rigshospitalet, Copenhagen, Denmark
12University Medical Center Groningen, Groningen, Netherlands
13University of California San Diego, La Jolla, CA, USA
14Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA
15Cliniques Universitaires Saint‐Luc, Université Catholique de Louvain, Brussels, Belgium
16Skåne University Hospital, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, Germany
18University of Utah, Salt Lake City, UT, USA
19University of South Florida, Tampa, FL, USA
20University of Arkansas for Medical Sciences, AR, USA
21University Hospital Southampton NHS Foundation Trust; Southampton, UK
22Cambridge University - Addenbrooke’s Hospital, Cambridge, UK
23University of Texas Health Science Center at Houston, Houston, TX, USA
24Phoenix Children’s Hospital, Phoenix USA
25Bloodworks Northwest, Seattle, WA
26WNY BloodCare, NY, USA
27Los Angeles Orthopedic 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, The Netherlands/ uniQure Inc. Lexington, MA, USA
31University Hospital Frankfurt, Frankfurt, Germany
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, M.D., 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, P.D30, 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, The Netherlands
5Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy
6Universitair Medisch Centrum Utrecht, Netherlands
7Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium
8Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands
9The National Coagulation Centre, St James’s Hospital, Dublin, Ireland
10Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, UK
11Rigshospitalet, Copenhagen, Denmark
12University Medical Center Groningen, Groningen, Netherlands
13University of California San Diego, La Jolla, CA, USA
14Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA
15Cliniques Universitaires Saint‐Luc, Université Catholique de Louvain, Brussels, Belgium
16Skåne University Hospital, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, Germany
18University of Utah, Salt Lake City, UT, USA
19University of South Florida, Tampa, FL, USA
20University of Arkansas for Medical Sciences, AR, USA
21University Hospital Southampton NHS Foundation Trust; Southampton, UK
22Cambridge University - Addenbrooke’s Hospital, Cambridge, UK
23University of Texas Health Science Center at Houston, Houston, TX, USA
24Phoenix Children’s Hospital, Phoenix USA
25Bloodworks Northwest, Seattle, WA
26WNY BloodCare, NY, USA
27Los Angeles Orthopedic 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, The Netherlands/ uniQure Inc. Lexington, MA, USA
31University Hospital Frankfurt, Frankfurt, Germany