Mga Highlight Mula sa Ika-62 na Taunang ASH ng Pagpupulong at Paglalahad
Unang Data Mula sa Phase 3 HOPE-B Gene Therapy Trial: Kahusayan at Kaligtasan ng Etranacogene Dezaparvovec (variant ng AAV5-Padua hFIX; AMT-061) sa Mga Matatanda na May Malubha o Katamtamang-Malubhang Hemophilia B Pinagamot na Hindi isinasaalang-alang ang Pre-Existing Anti-Capsid Neutralisasyon Mga Antibodies
Steven W. Pipa, 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 at Wolfgang A. Miesbach, MD, PhD38
1University of Michigan, Ann Arbor, MI
2Oregon Health & Science University, Portland, OR
3Unibersidad ng North Carolina, Chapel Hill, NC
4Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands
5Azienda Ospedaliera Universitaria Careggi, Florence, Italya
6Oregon Health & Science University, Portland
7Vrije Universiteit Medical Center, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Netherlands
9Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands
10Kagawaran ng Vascular Medicine at Haemostasis at Haemophilia Center, University Hospitals Leuven, Leuven, Belgium
11Amsterdam University Medical Center, University of Amsterdam, Amsterdam, Netherlands
12Ang National Coagulation Center, St James Hospital, Dublin, Ireland
13Royal London Haemophilia Center, Barts at London School of Medicine and Dentistry, London, United Kingdom
14Rigshospitalet, Copenhagen, Denmark
15University Medical Center Groningen, Groningen, Netherlands
16Unibersidad ng California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, Children's Hospital Los Angeles, Los Angeles, CA
18Cliniques Universitaire Saint-Luc, Universite Catholique de Louvain, Brussels, Belgium
19Kagawaran ng Translational Medicine-Clinical Coagulation, Lund University, Malmo, Sweden
20Skane University Hospital, Malmo, Sweden
21Vivantes Klinikum im Friedrichshain, Berlin, Germany
22University of Utah, Siyudad ng Salt Lake, UT
23University of South Florida, Tampa, FL
24University of Arkansas para sa Medical Science, Little Rock, AR
25University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom
26Cambridge University – Addenbrooke's Hospital, Cambridge, United Kingdom
27University of Texas Health Science Center sa Houston, Houston, TX
28Phoenix Children's Hospital, Phoenix, AZ
29Bloodworks Hilagang Kanluran, Seattle, WA
30Hemophilia Center ng Western New York, New York
31Los Angeles Orthopaedic Hospital, Orthopaedic Hemophilia Treatment Center, Los Angeles, CA
32Anschutz Medical Campus, University of Colorado, School of Medicine, Hemophilia at Thrombosis Center, Aurora, CO
33Vanderbilt University Medical Center, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38University Hospital Frankfurt, Frankfurt, Alemanya
Steven W. Pipa, 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 at Wolfgang A. Miesbach, MD, PhD38
1University of Michigan, Ann Arbor, MI
2Oregon Health & Science University, Portland, OR
3Unibersidad ng North Carolina, Chapel Hill, NC
4Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands
5Azienda Ospedaliera Universitaria Careggi, Florence, Italya
6Oregon Health & Science University, Portland
7Vrije Universiteit Medical Center, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Netherlands
9Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands
10Kagawaran ng Vascular Medicine at Haemostasis at Haemophilia Center, University Hospitals Leuven, Leuven, Belgium
11Amsterdam University Medical Center, University of Amsterdam, Amsterdam, Netherlands
12Ang National Coagulation Center, St James Hospital, Dublin, Ireland
13Royal London Haemophilia Center, Barts at London School of Medicine and Dentistry, London, United Kingdom
14Rigshospitalet, Copenhagen, Denmark
15University Medical Center Groningen, Groningen, Netherlands
16Unibersidad ng California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, Children's Hospital Los Angeles, Los Angeles, CA
18Cliniques Universitaire Saint-Luc, Universite Catholique de Louvain, Brussels, Belgium
19Kagawaran ng Translational Medicine-Clinical Coagulation, Lund University, Malmo, Sweden
20Skane University Hospital, Malmo, Sweden
21Vivantes Klinikum im Friedrichshain, Berlin, Germany
22University of Utah, Siyudad ng Salt Lake, UT
23University of South Florida, Tampa, FL
24University of Arkansas para sa Medical Science, Little Rock, AR
25University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom
26Cambridge University – Addenbrooke's Hospital, Cambridge, United Kingdom
27University of Texas Health Science Center sa Houston, Houston, TX
28Phoenix Children's Hospital, Phoenix, AZ
29Bloodworks Hilagang Kanluran, Seattle, WA
30Hemophilia Center ng Western New York, New York
31Los Angeles Orthopaedic Hospital, Orthopaedic Hemophilia Treatment Center, Los Angeles, CA
32Anschutz Medical Campus, University of Colorado, School of Medicine, Hemophilia at Thrombosis Center, Aurora, CO
33Vanderbilt University Medical Center, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38University Hospital Frankfurt, Frankfurt, Alemanya