Punti Ewlenin Mit-62 Laqgħa u Espożizzjoni Annwali tal-ASH
L-Ewwel Dejta Mill-Prova tat-Terapija tal-Ġeni HOPE-B tal-Fażi 3: Effikaċja u Sigurtà ta 'Etranacogene Dezaparvovec (varjant AAV5-Padova hFIX; AMT-061) f'Adulti b'Emofilja B Severa jew Moderata-Severa Ittrattata Irrispettivament minn Newtralizzanti Anti-Kapsidi Pre-Eżistenti Antikorpi
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, Michael 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. Lumi, 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 u Wolfgang A. Miesbach, MD, PhD38
1Università ta 'Michigan, Ann Arbor, MI
2Oregon Health & Science University, Portland, OR
3Università ta 'North Carolina, Chapel Hill, NC
4Erasmus MC, Ċentru Mediku Universitarju Rotterdam, Rotterdam, l-Olanda
5Azienda Ospedaliera Universitaria Careggi, Firenze, l-Italja
6Oregon Health & Science University, Portland
7Ċentru Mediku Vrije Universiteit, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, l-Olanda
9Van Creveldkliniek, Ċentru Mediku Universitarju Utrecht, Utrecht, l-Olanda
10Dipartiment tal-Mediċina Vaskulari u l-Emostasi u ċ-Ċentru tal-Emofilja, Sptarijiet Universitarji Leuven, Leuven, il-Belġju
11Ċentri Mediċi ta 'l-Università ta' Amsterdam, Università ta 'Amsterdam, Amsterdam, l-Olanda
12Iċ-Ċentru Nazzjonali tal-Koagulazzjoni, l-Isptar St James, Dublin, l-Irlanda
13Royal London Haemophilia Centre, Barts u l-London School of Medicine and Dentistry, Londra, ir-Renju Unit
14Rigshospitalet, Kopenħagen, id-Danimarka
15Ċentru Mediku Universitarju Groningen, Groningen, l-Olanda
16Università ta 'California San Diego, La Jolla, CA
17Università tal-Kalifornja tan-Nofsinhar Keck Iskola tal-Mediċina, l-Isptar tat-Tfal Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Brussell, il-Belġju
19Dipartiment tal-Mediċina Traslazzjonali-Koagulazzjoni Klinika, Lund University, Malmo, l-Iżvezja
20Skane University Hospital, Malmo, l-Iżvezja
21Vivantes Klinikum im Friedrichshain, Berlin, il-Ġermanja
22Università ta 'Utah, Salt Lake City, UT
23Università ta ’South Florida, Tampa, FL
24Università ta 'Arkansas għax-Xjenzi Mediċi, Little Rock, AR
25Isptar Universitarju Southampton NHS Foundation Trust, Southampton, ir-Renju Unit
26Cambridge University – Addenbrooke's Hospital, Cambridge, ir-Renju Unit
27Ċentru tax-Xjenza tas-Saħħa tal-Università ta 'Texas fi Houston, Houston, TX
28Isptar tat-Tfal ta 'Phoenix, Phoenix, AZ
29Bloodworks Majjistral, Seattle, WA
30Ċentru tal-Emofilja tal-Punent ta 'New York, New York
31Isptar Ortopediku ta 'Los Angeles, Ċentru ta' Trattament ta 'Emofilja Ortopedika, Los Angeles, CA
32Kampus Mediku Anschutz, Università ta 'Colorado, Iskola tal-Mediċina, Ċentru ta' Emofilja u Trombożi, Aurora, CO
33Ċentru Mediku tal-Università Vanderbilt, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38Sptar Universitarju ta ’Frankfurt, Frankfurt, il-Ġermanja
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, Michael 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. Lumi, 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 u Wolfgang A. Miesbach, MD, PhD38
1Università ta 'Michigan, Ann Arbor, MI
2Oregon Health & Science University, Portland, OR
3Università ta 'North Carolina, Chapel Hill, NC
4Erasmus MC, Ċentru Mediku Universitarju Rotterdam, Rotterdam, l-Olanda
5Azienda Ospedaliera Universitaria Careggi, Firenze, l-Italja
6Oregon Health & Science University, Portland
7Ċentru Mediku Vrije Universiteit, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, l-Olanda
9Van Creveldkliniek, Ċentru Mediku Universitarju Utrecht, Utrecht, l-Olanda
10Dipartiment tal-Mediċina Vaskulari u l-Emostasi u ċ-Ċentru tal-Emofilja, Sptarijiet Universitarji Leuven, Leuven, il-Belġju
11Ċentri Mediċi ta 'l-Università ta' Amsterdam, Università ta 'Amsterdam, Amsterdam, l-Olanda
12Iċ-Ċentru Nazzjonali tal-Koagulazzjoni, l-Isptar St James, Dublin, l-Irlanda
13Royal London Haemophilia Centre, Barts u l-London School of Medicine and Dentistry, Londra, ir-Renju Unit
14Rigshospitalet, Kopenħagen, id-Danimarka
15Ċentru Mediku Universitarju Groningen, Groningen, l-Olanda
16Università ta 'California San Diego, La Jolla, CA
17Università tal-Kalifornja tan-Nofsinhar Keck Iskola tal-Mediċina, l-Isptar tat-Tfal Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Brussell, il-Belġju
19Dipartiment tal-Mediċina Traslazzjonali-Koagulazzjoni Klinika, Lund University, Malmo, l-Iżvezja
20Skane University Hospital, Malmo, l-Iżvezja
21Vivantes Klinikum im Friedrichshain, Berlin, il-Ġermanja
22Università ta 'Utah, Salt Lake City, UT
23Università ta ’South Florida, Tampa, FL
24Università ta 'Arkansas għax-Xjenzi Mediċi, Little Rock, AR
25Isptar Universitarju Southampton NHS Foundation Trust, Southampton, ir-Renju Unit
26Cambridge University – Addenbrooke's Hospital, Cambridge, ir-Renju Unit
27Ċentru tax-Xjenza tas-Saħħa tal-Università ta 'Texas fi Houston, Houston, TX
28Isptar tat-Tfal ta 'Phoenix, Phoenix, AZ
29Bloodworks Majjistral, Seattle, WA
30Ċentru tal-Emofilja tal-Punent ta 'New York, New York
31Isptar Ortopediku ta 'Los Angeles, Ċentru ta' Trattament ta 'Emofilja Ortopedika, Los Angeles, CA
32Kampus Mediku Anschutz, Università ta 'Colorado, Iskola tal-Mediċina, Ċentru ta' Emofilja u Trombożi, Aurora, CO
33Ċentru Mediku tal-Università Vanderbilt, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38Sptar Universitarju ta ’Frankfurt, Frankfurt, il-Ġermanja