Sorotan Dari Mesyuarat dan Pameran Tahunan ASH ke-62
Data Pertama Dari Percubaan Terapi Gen Fasa 3 HOPE-B: Keberkesanan dan Keselamatan Etranacogene Dezaparvovec (varian AAV5-Padua hFIX; AMT-061) pada Orang Dewasa Dengan Hemofilia B yang teruk atau sederhana yang diubati tanpa mengira Neutralisasi Anti-Kapsid Pra-Sedia Ada 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 Young, 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
1Universiti Michigan, Ann Arbor, MI
2Universiti Kesihatan & Sains Oregon, Portland, OR
3Universiti Carolina Utara, Chapel Hill, NC
4Erasmus MC, Pusat Perubatan Universiti Rotterdam, Rotterdam, Belanda
5Azienda Ospedaliera Universitaria Careggi, Florence, Itali
6Universiti Kesihatan & Sains Oregon, Portland
7Pusat Perubatan Vrije Universiteit, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Belanda
9Van Creveldkliniek, Pusat Perubatan Universiti Utrecht, Utrecht, Belanda
10Jabatan Perubatan Vaskular dan Pusat Haemostasis dan Haemophilia, Hospital Universiti Leuven, Leuven, Belgium
11Pusat Perubatan Universiti Amsterdam, Universiti Amsterdam, Amsterdam, Belanda
12Pusat Pembekuan Nasional, Hospital St James, Dublin, Ireland
13Pusat London Haemophilia, Barts dan London School of Medicine and Dentistry, London, United Kingdom
14Rigshospitalet, Copenhagen, Denmark
15Pusat Perubatan Universiti Groningen, Groningen, Belanda
16Universiti California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, Hospital Kanak-kanak Los Angeles, Los Angeles, CA
18Cliniques Universitaire Saint-Luc, Universite Catholique de Louvain, Brussels, Belgium
19Jabatan Perubatan Translasional-Pembekuan Klinikal, Universiti Lund, Malmo, Sweden
20Hospital Universiti Skane, Malmo, Sweden
21Vivantes Klinikum im Friedrichshain, Berlin, Jerman
22Universiti Utah, Salt Lake City, UT
23University of South Florida, Tampa, FL
24Universiti Arkansas untuk Sains Perubatan, Little Rock, AR
25Hospital Universiti Southampton NHS Foundation Trust, Southampton, United Kingdom
26Universiti Cambridge – Hospital Addenbrooke, Cambridge, United Kingdom
27Pusat Sains Kesihatan Universiti Texas di Houston, Houston, TX
28Hospital Kanak-kanak Phoenix, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Pusat Hemofilia New York Barat, New York
31Hospital Orthopedik Los Angeles, Pusat Rawatan Hemofilia Ortopedik, Los Angeles, CA
32Kampus Perubatan Anschutz, University of Colorado, Sekolah Perubatan, Pusat Hemofilia dan Trombosis, Aurora, CO
33Pusat Perubatan Universiti Vanderbilt, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38Hospital Universiti 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 Young, 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
1Universiti Michigan, Ann Arbor, MI
2Universiti Kesihatan & Sains Oregon, Portland, OR
3Universiti Carolina Utara, Chapel Hill, NC
4Erasmus MC, Pusat Perubatan Universiti Rotterdam, Rotterdam, Belanda
5Azienda Ospedaliera Universitaria Careggi, Florence, Itali
6Universiti Kesihatan & Sains Oregon, Portland
7Pusat Perubatan Vrije Universiteit, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Belanda
9Van Creveldkliniek, Pusat Perubatan Universiti Utrecht, Utrecht, Belanda
10Jabatan Perubatan Vaskular dan Pusat Haemostasis dan Haemophilia, Hospital Universiti Leuven, Leuven, Belgium
11Pusat Perubatan Universiti Amsterdam, Universiti Amsterdam, Amsterdam, Belanda
12Pusat Pembekuan Nasional, Hospital St James, Dublin, Ireland
13Pusat London Haemophilia, Barts dan London School of Medicine and Dentistry, London, United Kingdom
14Rigshospitalet, Copenhagen, Denmark
15Pusat Perubatan Universiti Groningen, Groningen, Belanda
16Universiti California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, Hospital Kanak-kanak Los Angeles, Los Angeles, CA
18Cliniques Universitaire Saint-Luc, Universite Catholique de Louvain, Brussels, Belgium
19Jabatan Perubatan Translasional-Pembekuan Klinikal, Universiti Lund, Malmo, Sweden
20Hospital Universiti Skane, Malmo, Sweden
21Vivantes Klinikum im Friedrichshain, Berlin, Jerman
22Universiti Utah, Salt Lake City, UT
23University of South Florida, Tampa, FL
24Universiti Arkansas untuk Sains Perubatan, Little Rock, AR
25Hospital Universiti Southampton NHS Foundation Trust, Southampton, United Kingdom
26Universiti Cambridge – Hospital Addenbrooke, Cambridge, United Kingdom
27Pusat Sains Kesihatan Universiti Texas di Houston, Houston, TX
28Hospital Kanak-kanak Phoenix, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Pusat Hemofilia New York Barat, New York
31Hospital Orthopedik Los Angeles, Pusat Rawatan Hemofilia Ortopedik, Los Angeles, CA
32Kampus Perubatan Anschutz, University of Colorado, Sekolah Perubatan, Pusat Hemofilia dan Trombosis, Aurora, CO
33Pusat Perubatan Universiti Vanderbilt, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38Hospital Universiti Frankfurt, Frankfurt, Jerman