Sorotan Dari Kongres Tahunan ke-14 EAHAD
Keberkesanan dan Keselamatan Etranacogene Dezaparvovec pada Orang Dewasa Dengan Hemofilia B yang teruk atau sederhana: Data Pertama Dari Percubaan Terapi Gen Tahap-B Fasa 3
Steven W Pipe, MD1, Michael Recht, MD, PhD2, Kunci S Nigel, 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, Amerika Syarikat
2Universiti Kesihatan & Sains Oregon, Portland, OR, USA
3University of North Carolina, Chapel Hill, NC, Amerika Syarikat
4Erasmus MC, Pusat Perubatan Universiti, Rotterdam, Belanda
5Pusat Gangguan Pendarahan dan Pembekuan, Hospital Universiti Careggi, Florence, Itali
6Universitair Medisch Centrum Utrecht, Belanda
7Jabatan Perubatan Vaskular dan Pusat Haemostasis dan Haemophilia, Hospital Universiti Leuven, Leuven, Belgium
8Jabatan Perubatan Vaskular, Sains Kardiovaskular Amsterdam, Pusat Perubatan Universiti Amsterdam, Universiti Amsterdam, Amsterdam, Belanda
9Pusat Pembekuan Nasional, St James's Hospital, Dublin, Ireland
10Pusat Haemofilia Royal London, Barts dan London School of Medicine and Dentistry, London, UK
11Rigshospitalet, Copenhagen, Denmark
12Pusat Perubatan Universiti Groningen, Groningen, Belanda
13University of California San Diego, La Jolla, CA, Amerika Syarikat
14Hospital Kanak-kanak Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, Amerika Syarikat
15Cliniques Universitaires Saint ‐ Luc, Université Catholique de Louvain, Brussels, Belgium
16Hospital Universiti Skåne, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, Jerman
18University of Utah, Salt Lake City, UT, Amerika Syarikat
19University of South Florida, Tampa, FL, Amerika Syarikat
20University of Arkansas untuk Sains Perubatan, AR, Amerika Syarikat
21Universiti Hospital Southampton NHS Foundation Trust; Southampton, UK
22Universiti Cambridge - Hospital Addenbrooke, Cambridge, UK
23Pusat Sains Kesihatan Universiti Texas di Houston, Houston, TX, Amerika Syarikat
24Hospital Kanak-kanak Phoenix, Phoenix Amerika Syarikat
25Bloodworks Northwest, Seattle, WA
26WNY BloodCare, NY, Amerika Syarikat
27Hospital Orthopedik Los Angeles, Pusat Rawatan Hemofilia Ortopedik, Los Angeles, CA, Amerika Syarikat
28Pusat Hemofilia dan Trombosis Universiti Colorado, Kampus Perubatan Anschutz, Aurora, CO
29Pusat Perubatan Universiti Vanderbilt, Nashville, TN, Amerika Syarikat
30uniQure BV, Amsterdam, Belanda / uniQure Inc. Lexington, MA, Amerika Syarikat
31Hospital Universiti Frankfurt, Frankfurt, Jerman
Steven W Pipe, MD1, Michael Recht, MD, PhD2, Kunci S Nigel, 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, Amerika Syarikat
2Universiti Kesihatan & Sains Oregon, Portland, OR, USA
3University of North Carolina, Chapel Hill, NC, Amerika Syarikat
4Erasmus MC, Pusat Perubatan Universiti, Rotterdam, Belanda
5Pusat Gangguan Pendarahan dan Pembekuan, Hospital Universiti Careggi, Florence, Itali
6Universitair Medisch Centrum Utrecht, Belanda
7Jabatan Perubatan Vaskular dan Pusat Haemostasis dan Haemophilia, Hospital Universiti Leuven, Leuven, Belgium
8Jabatan Perubatan Vaskular, Sains Kardiovaskular Amsterdam, Pusat Perubatan Universiti Amsterdam, Universiti Amsterdam, Amsterdam, Belanda
9Pusat Pembekuan Nasional, St James's Hospital, Dublin, Ireland
10Pusat Haemofilia Royal London, Barts dan London School of Medicine and Dentistry, London, UK
11Rigshospitalet, Copenhagen, Denmark
12Pusat Perubatan Universiti Groningen, Groningen, Belanda
13University of California San Diego, La Jolla, CA, Amerika Syarikat
14Hospital Kanak-kanak Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, Amerika Syarikat
15Cliniques Universitaires Saint ‐ Luc, Université Catholique de Louvain, Brussels, Belgium
16Hospital Universiti Skåne, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, Jerman
18University of Utah, Salt Lake City, UT, Amerika Syarikat
19University of South Florida, Tampa, FL, Amerika Syarikat
20University of Arkansas untuk Sains Perubatan, AR, Amerika Syarikat
21Universiti Hospital Southampton NHS Foundation Trust; Southampton, UK
22Universiti Cambridge - Hospital Addenbrooke, Cambridge, UK
23Pusat Sains Kesihatan Universiti Texas di Houston, Houston, TX, Amerika Syarikat
24Hospital Kanak-kanak Phoenix, Phoenix Amerika Syarikat
25Bloodworks Northwest, Seattle, WA
26WNY BloodCare, NY, Amerika Syarikat
27Hospital Orthopedik Los Angeles, Pusat Rawatan Hemofilia Ortopedik, Los Angeles, CA, Amerika Syarikat
28Pusat Hemofilia dan Trombosis Universiti Colorado, Kampus Perubatan Anschutz, Aurora, CO
29Pusat Perubatan Universiti Vanderbilt, Nashville, TN, Amerika Syarikat
30uniQure BV, Amsterdam, Belanda / uniQure Inc. Lexington, MA, Amerika Syarikat
31Hospital Universiti Frankfurt, Frankfurt, Jerman