Sorotan Dari Kongres Tahunan ke-14 EAHAD
Khasiat dan Keamanan Etranacogene Dezaparvovec pada Orang Dewasa Dengan Hemofilia B Parah atau Sedang-parah: Data Pertama Dari Percobaan Terapi Gen Harapan-B Tahap 3
Steven W Pipa, MD1, Michael Recht, MD, PhD2, Kunci Nigel S, 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 Muda, MD14, Cedric Hermans, MD15, Jan Astermark, MD, PhD16, Robert Klamroth, MD, PhD17, Richard S Lemon, 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
1Universitas Michigan, Ann Arbor, MI, AS
2Universitas Kesehatan & Sains Oregon, Portland, OR, AS
3Universitas Carolina Utara, Chapel Hill, NC, AS
4Erasmus MC, University Medical Center, Rotterdam, Belanda
5Pusat Gangguan Pendarahan dan Koagulasi, Rumah Sakit Universitas Careggi, Florence, Italia
6Universitair Medisch Centrum Utrecht, Belanda
7Departemen Kedokteran Vaskular dan Pusat Hemostasis dan Hemofilia, Rumah Sakit Universitas Leuven, Leuven, Belgia
8Departemen Kedokteran Vaskular, Ilmu Kardiovaskular Amsterdam, Pusat Medis Universitas Amsterdam, Universitas Amsterdam, Amsterdam, Belanda
9Pusat Koagulasi Nasional, Rumah Sakit St James, Dublin, Irlandia
10Royal London Haemophilia Center, Barts and the London School of Medicine and Dentistry, London, UK
11Rigshospitalet, Kopenhagen, Denmark
12Pusat Medis Universitas Groningen, Groningen, Belanda
13Universitas California San Diego, La Jolla, CA, AS
14Rumah Sakit Anak Los Angeles, Fakultas Kedokteran Universitas California Selatan Keck, Los Angeles, CA, AS
15Cliniques Universitaires Saint ‐ Luc, Université Catholique de Louvain, Brussels, Belgia
16Rumah Sakit Universitas Skåne, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, Jerman
18Universitas Utah, Salt Lake City, UT, AS
19Universitas Florida Selatan, Tampa, FL, AS
20University of Arkansas untuk Ilmu Kedokteran, AR, AS
21Rumah Sakit Universitas Southampton NHS Foundation Trust; Southampton, Inggris
22Universitas Cambridge - Rumah Sakit Addenbrooke, Cambridge, Inggris
23Pusat Ilmu Kesehatan Universitas Texas di Houston, Houston, TX, AS
24Rumah Sakit Anak Phoenix, Phoenix USA
25Bloodworks Northwest, Seattle, WA
26WNY BloodCare, NY, AS
27Rumah Sakit Ortopedi Los Angeles, Pusat Perawatan Hemofilia Ortopedi, Los Angeles, CA, AS
28Pusat Hemofilia dan Trombosis Universitas Colorado, Kampus Medis Anschutz, Aurora, CO
29Pusat Medis Universitas Vanderbilt, Nashville, TN, AS
30uniQure BV, Amsterdam, Belanda / uniQure Inc. Lexington, MA, USA
31Rumah Sakit Universitas Frankfurt, Frankfurt, Jerman
Steven W Pipa, MD1, Michael Recht, MD, PhD2, Kunci Nigel S, 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 Muda, MD14, Cedric Hermans, MD15, Jan Astermark, MD, PhD16, Robert Klamroth, MD, PhD17, Richard S Lemon, 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
1Universitas Michigan, Ann Arbor, MI, AS
2Universitas Kesehatan & Sains Oregon, Portland, OR, AS
3Universitas Carolina Utara, Chapel Hill, NC, AS
4Erasmus MC, University Medical Center, Rotterdam, Belanda
5Pusat Gangguan Pendarahan dan Koagulasi, Rumah Sakit Universitas Careggi, Florence, Italia
6Universitair Medisch Centrum Utrecht, Belanda
7Departemen Kedokteran Vaskular dan Pusat Hemostasis dan Hemofilia, Rumah Sakit Universitas Leuven, Leuven, Belgia
8Departemen Kedokteran Vaskular, Ilmu Kardiovaskular Amsterdam, Pusat Medis Universitas Amsterdam, Universitas Amsterdam, Amsterdam, Belanda
9Pusat Koagulasi Nasional, Rumah Sakit St James, Dublin, Irlandia
10Royal London Haemophilia Center, Barts and the London School of Medicine and Dentistry, London, UK
11Rigshospitalet, Kopenhagen, Denmark
12Pusat Medis Universitas Groningen, Groningen, Belanda
13Universitas California San Diego, La Jolla, CA, AS
14Rumah Sakit Anak Los Angeles, Fakultas Kedokteran Universitas California Selatan Keck, Los Angeles, CA, AS
15Cliniques Universitaires Saint ‐ Luc, Université Catholique de Louvain, Brussels, Belgia
16Rumah Sakit Universitas Skåne, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, Jerman
18Universitas Utah, Salt Lake City, UT, AS
19Universitas Florida Selatan, Tampa, FL, AS
20University of Arkansas untuk Ilmu Kedokteran, AR, AS
21Rumah Sakit Universitas Southampton NHS Foundation Trust; Southampton, Inggris
22Universitas Cambridge - Rumah Sakit Addenbrooke, Cambridge, Inggris
23Pusat Ilmu Kesehatan Universitas Texas di Houston, Houston, TX, AS
24Rumah Sakit Anak Phoenix, Phoenix USA
25Bloodworks Northwest, Seattle, WA
26WNY BloodCare, NY, AS
27Rumah Sakit Ortopedi Los Angeles, Pusat Perawatan Hemofilia Ortopedi, Los Angeles, CA, AS
28Pusat Hemofilia dan Trombosis Universitas Colorado, Kampus Medis Anschutz, Aurora, CO
29Pusat Medis Universitas Vanderbilt, Nashville, TN, AS
30uniQure BV, Amsterdam, Belanda / uniQure Inc. Lexington, MA, USA
31Rumah Sakit Universitas Frankfurt, Frankfurt, Jerman