Buaicphointí Ón 62ú Cruinniú Bliantúil agus Taispeántas ASH
Na Chéad Sonraí Ó Thriail Teiripe Géine HOPE-B Céim 3: Éifeachtúlacht agus Sábháilteacht Etranacogene Dezaparvovec (leagan AAV5-Padua hFIX; AMT-061) i measc Daoine Fásta a bhfuil cóireáil hemophilia B tromchúiseach nó measartha trom orthu, is cuma cén Neodrú Frith-Capsid atá ann cheana Antasubstaintí
Steven W. Píob, MD1, Michael Recht, MD, PhD2, Nigel S. Eochair, 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 Óg, 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 agus Wolfgang A. Miesbach, MD, PhD38
1Ollscoil Michigan, Ann Arbor, MI
2Ollscoil Sláinte & Eolaíochta Oregon, Portland, NÓ
3Ollscoil Carolina Thuaidh, Chapel Hill, NC
4Erasmus MC, Ionad Leighis na hOllscoile Rotterdam, Rotterdam, an Ísiltír
5Azienda Ospedaliera Universitaria Careggi, Florence, an Iodáil
6Ollscoil Sláinte & Eolaíochta Oregon, Portland
7Ionad Leighis Vrije Universiteit, Amstardam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, An Ísiltír
9Van Creveldkliniek, Ionad Leighis na hOllscoile Utrecht, Utrecht, An Ísiltír
10Roinn an Leighis Leigheas Soithíocha agus Haemostasis agus Ionad Haemafilia, Ospidéil Ollscoile Leuven, Leuven, an Bheilg
11Ionaid Leighis Ollscoil Amstardam, Ollscoil Amstardam, Amstardam, an Ísiltír
12An tIonad Náisiúnta Téachta, Ospidéal San Séamas, Baile Átha Cliath, Éire
13Ionad Haemophilia Ríoga Londain, Barts agus Scoil Leighis agus Fiaclóireachta Londain, Londain, an Ríocht Aontaithe
14Rigshospitalet, Cóbanhávan, an Danmhairg
15Ionad Leighis na hOllscoile Groningen, Groningen, An Ísiltír
16Ollscoil California San Diego, La Jolla, CA.
17Scoil an Leighis Ollscoil Southern California Keck, Ospidéal na Leanaí Los Angeles, Los Angeles, CA.
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, an Bhruiséil, an Bheilg
19Roinn an Leighis Aistriúcháin-Téachtadh Cliniciúil, Ollscoil Lund, Malmo, an tSualainn
20Ospidéal Ollscoile Skane, Malmo, an tSualainn
21Vivantes Klinikum im Friedrichshain, Beirlín, an Ghearmáin
22Ollscoil Utah, Salt Lake City, UT
23Ollscoil Florida Theas, Tampa, FL
24Ollscoil Arkansas um Eolaíochtaí Leighis, Little Rock, AR
25Iontaobhas Fondúireachta NHS Ospidéal na hOllscoile, Southampton, An Ríocht Aontaithe
26Ollscoil Cambridge - Ospidéal Addenbrooke, Cambridge, an Ríocht Aontaithe
27Ionad Eolaíochta Sláinte Ollscoil Texas ag Houston, Houston, TX
28Ospidéal Leanaí an Fhionnuisce, an Fhionnuisce, AZ
29Bloodworks Iarthuaisceart, Seattle, WA
30Ionad Hemophilia in Iarthar Nua Eabhrac, Nua Eabhrac
31Ospidéal Ortaipéideach Los Angeles, Ionad Cóireála Ortaipéideach Ortaipéideach, Los Angeles, CA.
32Campas Leighis Anschutz, Ollscoil Colorado, Scoil an Leighis, Ionad Hemophilia agus Thrombosis, Aurora, CO
33Ionad Leighis Ollscoil Vanderbilt, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38Ospidéal na hOllscoile Frankfurt, Frankfurt, an Ghearmáin
Steven W. Píob, MD1, Michael Recht, MD, PhD2, Nigel S. Eochair, 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 Óg, 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 agus Wolfgang A. Miesbach, MD, PhD38
1Ollscoil Michigan, Ann Arbor, MI
2Ollscoil Sláinte & Eolaíochta Oregon, Portland, NÓ
3Ollscoil Carolina Thuaidh, Chapel Hill, NC
4Erasmus MC, Ionad Leighis na hOllscoile Rotterdam, Rotterdam, an Ísiltír
5Azienda Ospedaliera Universitaria Careggi, Florence, an Iodáil
6Ollscoil Sláinte & Eolaíochta Oregon, Portland
7Ionad Leighis Vrije Universiteit, Amstardam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, An Ísiltír
9Van Creveldkliniek, Ionad Leighis na hOllscoile Utrecht, Utrecht, An Ísiltír
10Roinn an Leighis Leigheas Soithíocha agus Haemostasis agus Ionad Haemafilia, Ospidéil Ollscoile Leuven, Leuven, an Bheilg
11Ionaid Leighis Ollscoil Amstardam, Ollscoil Amstardam, Amstardam, an Ísiltír
12An tIonad Náisiúnta Téachta, Ospidéal San Séamas, Baile Átha Cliath, Éire
13Ionad Haemophilia Ríoga Londain, Barts agus Scoil Leighis agus Fiaclóireachta Londain, Londain, an Ríocht Aontaithe
14Rigshospitalet, Cóbanhávan, an Danmhairg
15Ionad Leighis na hOllscoile Groningen, Groningen, An Ísiltír
16Ollscoil California San Diego, La Jolla, CA.
17Scoil an Leighis Ollscoil Southern California Keck, Ospidéal na Leanaí Los Angeles, Los Angeles, CA.
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, an Bhruiséil, an Bheilg
19Roinn an Leighis Aistriúcháin-Téachtadh Cliniciúil, Ollscoil Lund, Malmo, an tSualainn
20Ospidéal Ollscoile Skane, Malmo, an tSualainn
21Vivantes Klinikum im Friedrichshain, Beirlín, an Ghearmáin
22Ollscoil Utah, Salt Lake City, UT
23Ollscoil Florida Theas, Tampa, FL
24Ollscoil Arkansas um Eolaíochtaí Leighis, Little Rock, AR
25Iontaobhas Fondúireachta NHS Ospidéal na hOllscoile, Southampton, An Ríocht Aontaithe
26Ollscoil Cambridge - Ospidéal Addenbrooke, Cambridge, an Ríocht Aontaithe
27Ionad Eolaíochta Sláinte Ollscoil Texas ag Houston, Houston, TX
28Ospidéal Leanaí an Fhionnuisce, an Fhionnuisce, AZ
29Bloodworks Iarthuaisceart, Seattle, WA
30Ionad Hemophilia in Iarthar Nua Eabhrac, Nua Eabhrac
31Ospidéal Ortaipéideach Los Angeles, Ionad Cóireála Ortaipéideach Ortaipéideach, Los Angeles, CA.
32Campas Leighis Anschutz, Ollscoil Colorado, Scoil an Leighis, Ionad Hemophilia agus Thrombosis, Aurora, CO
33Ionad Leighis Ollscoil Vanderbilt, Nashville, TN
34uniQure Inc, Lexington, MA
35uniQure, Lexington, MA
36uniQure Inc, Lexington, MA
37Uniqure Inc, Lexington, MA
38Ospidéal na hOllscoile Frankfurt, Frankfurt, an Ghearmáin