Sorotan Dari Pertemuan Tahunan dan Eksposisi ASH ke-62
Data Pertama Dari Percobaan Terapi Gen HOPE-B Tahap 3: Khasiat dan Keamanan Etranacogene Dezaparvovec (varian AAV5-Padua hFIX; AMT-061) pada Orang Dewasa Dengan Hemofilia B Parah atau Sedang-Parah Yang Diobati Terlepas dari Penetralan Anti-Kapsid Yang Sudah Ada Sebelumnya Antibodi
Steven W.Pipe, MD1, Michael Recht, MD, PhD2, Nigel S.Key, MD3, Frank WG Leebeek, MD, PhD4, Giancarlo Castaman, MD5, Susan U.Lattimore, RN6, Paul Van Der Valk7,8,9, Kathelijne Peerlinck, MD, PhD10, Michiel Coppens, MD11, Niamh O'Connell, MD, PhD12, John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH13, Peter Kampmann, MD14, Karina Meijer, MD, PhD15, Annette von Drygalski, MD, PharmD16, Guy Muda, MD17, Cedric Hermans, MD, MRCP, PhD18, Jan Astermark, MD, PhD19,20, Robert Klamroth, MD, PhD21, Richard S.Lemons, MD22, Nathan Visweshwar, MD23, Shelley Crary, MD, MS24, Rashid Kazmi, MBBS25, Emily Symington26, Miguel A. Escobar, MD27, Esteban Gomez, MD28, Rebecca Kruse-Jarres, MD29, Adam Kotowski, MD30, Doris Quon, MD, PhD31, Michael Wang, MD32, Allison P. Wheeler, MD33, Eileen K Sawyer, PhD34, Stephanie Verweij, BSc35, Valerie Colletta, MSc36, Naghmana Bajma, MD37, Robert Gut, MD, PhD36 dan Wolfgang A. Miesbach, MD, PhD38
1Universitas Michigan, Ann Arbor, MI
2Universitas Kesehatan & Sains Oregon, Portland, OR
3Universitas Carolina Utara, Chapel Hill, NC
4Erasmus MC, University Medical Center Rotterdam, Rotterdam, Belanda
5Azienda Ospedaliera Universitaria Careggi, Florence, Italia
6Universitas Kesehatan & Sains Oregon, Portland
7Pusat Medis Vrije Universiteit, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Belanda
9Van Creveldkliniek, Pusat Medis Universitas Utrecht, Utrecht, Belanda
10Departemen Kedokteran Vaskular dan Pusat Hemostasis dan Hemofilia, Rumah Sakit Universitas Leuven, Leuven, Belgia
11Pusat Medis Universitas Amsterdam, Universitas Amsterdam, Amsterdam, Belanda
12Pusat Koagulasi Nasional, Rumah Sakit St James, Dublin, Irlandia
13Royal London Haemophilia Center, Barts and the London School of Medicine and Dentistry, London, United Kingdom
14Rigshospitalet, Kopenhagen, Denmark
15Pusat Medis Universitas Groningen, Groningen, Belanda
16Universitas California San Diego, La Jolla, CA
17Fakultas Kedokteran Universitas Southern California Keck, Rumah Sakit Anak Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universitas Catholique de Louvain, Brussels, Belgia
19Departemen Translational Medicine-Clinical Coagulation, Lund University, Malmo, Swedia
20Rumah Sakit Universitas Skane, Malmo, Swedia
21Vivantes Klinikum im Friedrichshain, Berlin, Jerman
22Universitas Utah, Salt Lake City, UT
23University of South Florida, Tampa, FL
24University of Arkansas untuk Ilmu Kedokteran, Little Rock, AR
25Rumah Sakit Universitas Southampton NHS Foundation Trust, Southampton, Inggris Raya
26Universitas Cambridge – Rumah Sakit Addenbrooke, Cambridge, Britania Raya
27Pusat Ilmu Kesehatan Universitas Texas di Houston, Houston, TX
28Rumah Sakit Anak Phoenix, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Pusat Hemofilia di New York Barat, New York
31Rumah Sakit Ortopedi Los Angeles, Pusat Perawatan Hemofilia Ortopedi, Los Angeles, CA
32Kampus Medis Anschutz, Universitas Colorado, Sekolah Kedokteran, Pusat Hemofilia dan Trombosis, Aurora, CO
33Pusat Medis Universitas Vanderbilt, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38Rumah Sakit Universitas Frankfurt, Frankfurt, Jerman
Steven W.Pipe, MD1, Michael Recht, MD, PhD2, Nigel S.Key, MD3, Frank WG Leebeek, MD, PhD4, Giancarlo Castaman, MD5, Susan U.Lattimore, RN6, Paul Van Der Valk7,8,9, Kathelijne Peerlinck, MD, PhD10, Michiel Coppens, MD11, Niamh O'Connell, MD, PhD12, John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH13, Peter Kampmann, MD14, Karina Meijer, MD, PhD15, Annette von Drygalski, MD, PharmD16, Guy Muda, MD17, Cedric Hermans, MD, MRCP, PhD18, Jan Astermark, MD, PhD19,20, Robert Klamroth, MD, PhD21, Richard S.Lemons, MD22, Nathan Visweshwar, MD23, Shelley Crary, MD, MS24, Rashid Kazmi, MBBS25, Emily Symington26, Miguel A. Escobar, MD27, Esteban Gomez, MD28, Rebecca Kruse-Jarres, MD29, Adam Kotowski, MD30, Doris Quon, MD, PhD31, Michael Wang, MD32, Allison P. Wheeler, MD33, Eileen K Sawyer, PhD34, Stephanie Verweij, BSc35, Valerie Colletta, MSc36, Naghmana Bajma, MD37, Robert Gut, MD, PhD36 dan Wolfgang A. Miesbach, MD, PhD38
1Universitas Michigan, Ann Arbor, MI
2Universitas Kesehatan & Sains Oregon, Portland, OR
3Universitas Carolina Utara, Chapel Hill, NC
4Erasmus MC, University Medical Center Rotterdam, Rotterdam, Belanda
5Azienda Ospedaliera Universitaria Careggi, Florence, Italia
6Universitas Kesehatan & Sains Oregon, Portland
7Pusat Medis Vrije Universiteit, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Belanda
9Van Creveldkliniek, Pusat Medis Universitas Utrecht, Utrecht, Belanda
10Departemen Kedokteran Vaskular dan Pusat Hemostasis dan Hemofilia, Rumah Sakit Universitas Leuven, Leuven, Belgia
11Pusat Medis Universitas Amsterdam, Universitas Amsterdam, Amsterdam, Belanda
12Pusat Koagulasi Nasional, Rumah Sakit St James, Dublin, Irlandia
13Royal London Haemophilia Center, Barts and the London School of Medicine and Dentistry, London, United Kingdom
14Rigshospitalet, Kopenhagen, Denmark
15Pusat Medis Universitas Groningen, Groningen, Belanda
16Universitas California San Diego, La Jolla, CA
17Fakultas Kedokteran Universitas Southern California Keck, Rumah Sakit Anak Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universitas Catholique de Louvain, Brussels, Belgia
19Departemen Translational Medicine-Clinical Coagulation, Lund University, Malmo, Swedia
20Rumah Sakit Universitas Skane, Malmo, Swedia
21Vivantes Klinikum im Friedrichshain, Berlin, Jerman
22Universitas Utah, Salt Lake City, UT
23University of South Florida, Tampa, FL
24University of Arkansas untuk Ilmu Kedokteran, Little Rock, AR
25Rumah Sakit Universitas Southampton NHS Foundation Trust, Southampton, Inggris Raya
26Universitas Cambridge – Rumah Sakit Addenbrooke, Cambridge, Britania Raya
27Pusat Ilmu Kesehatan Universitas Texas di Houston, Houston, TX
28Rumah Sakit Anak Phoenix, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Pusat Hemofilia di New York Barat, New York
31Rumah Sakit Ortopedi Los Angeles, Pusat Perawatan Hemofilia Ortopedi, Los Angeles, CA
32Kampus Medis Anschutz, Universitas Colorado, Sekolah Kedokteran, Pusat Hemofilia dan Trombosis, Aurora, CO
33Pusat Medis Universitas Vanderbilt, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38Rumah Sakit Universitas Frankfurt, Frankfurt, Jerman