Punti Ewlenin Mill-14-il Kungress Annwali tal-EAHAD
L-Effikaċja u s-Sikurezza ta 'Etranacogene Dezaparvovec f'Adulti b'Hemofilja B severa jew moderata-severa: L-Ewwel Dejta Mill-Prova tal-Fażi 3 Hope-B Terapija tal-Ġeni
Steven W Pipe, MD1, Michael Recht, MD, PhD2, Nigel S Key, MBChB3, Frank WG Leebeek, MD4, Giancarlo Castaman, MD5, Susan U Lattimore, RN6, Paul van der Valk, MD6, Kathelijne Peerlinck, MD, PhD7, Michael Coppens, MD, PhD8, Niamh O'Connell, MB PhD9, K. John Pasi, MBChB, PhD10, Peter Kampmann, MD11, Karina Meijer, MD, PhD.12, Annette von Drygalski, MD, PharmD13, Guy Young, MD14, Cedric Hermans, MD15, Jan Astermark, MD, PhD16, Robert Klamroth, MD, PhD17, Richard S Lemons, MD, PhD18, Nathan Visweshwar, MD19, Shelley Crary, MD20, Rashid Kazmi, MBBS21, Emily Symington, MBChB22, Miguel A Escobar, MD23, Esteban Gomez, MD24, Rebecca Kruse-Jarres, MD, MPH25, Adam Kotowski, MD26, Doris Quon, MD, PhD27, Michael Wang, MD28, Allison P Wheeler, MD29, Eileen K Sawyer, PhD30, Stephanie Verweij30, Valerie Colletta, MSc30, Naghmana Bajwa, MD, Robert Gut, MD, PD30, Wolfgang Miesbach, MD, PhD31
1Università ta 'Michigan, Ann Arbor, MI, USA
2Oregon Health & Science University, Portland, OR, USA
3Università ta 'North Carolina, Chapel Hill, NC, USA
4Erasmus MC, Ċentru Mediku Universitarju, Rotterdam, l-Olanda
5Ċentru għal Disturbi ta ’Fsada u Koagulazzjoni, Sptar Universitarju Careggi, Firenze, l-Italja
6Universitair Medisch Centrum Utrecht, l-Olanda
7Dipartiment tal-Mediċina Vaskulari u l-Emostasi u ċ-Ċentru tal-Emofilja, Sptarijiet Universitarji Leuven, Leuven, il-Belġju
8Dipartiment tal-Mediċina Vaskulari, Xjenzi Kardjovaskulari ta 'Amsterdam, Ċentri Mediċi ta' l-Università ta 'Amsterdam, Università ta' Amsterdam, Amsterdam, l-Olanda
9Iċ-Ċentru Nazzjonali tal-Koagulazzjoni, l-Isptar St James, Dublin, l-Irlanda
10Royal London Haemophilia Centre, Barts u l-London School of Medicine and Dentistry, Londra, ir-Renju Unit
11Rigshospitalet, Kopenħagen, id-Danimarka
12Ċentru Mediku Universitarju Groningen, Groningen, l-Olanda
13Università ta 'California San Diego, La Jolla, CA, USA
14L-Isptar tat-Tfal Los Angeles, l-Iskola tal-Mediċina Keck ta 'l-Università ta' Southern California, Los Angeles, CA, l-Istati Uniti
15Cliniques Universitaires Saint ‐ Luc, Université Catholique de Louvain, Brussell, il-Belġju
16Sptar Universitarju Skåne, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, il-Ġermanja
18Università ta 'Utah, Salt Lake City, UT, USA
19Università ta 'South Florida, Tampa, FL, USA
20Università ta 'Arkansas għax-Xjenzi Mediċi, AR, USA
21Università ta 'l-Isptar Southampton NHS Foundation Trust; Southampton, ir-Renju Unit
22Università ta 'Cambridge - Addenbrooke's Hospital, Cambridge, ir-Renju Unit
23Ċentru tax-Xjenza tas-Saħħa tal-Università ta 'Texas fi Houston, Houston, TX, l-Istati Uniti
24Phoenix Children Hospital, Phoenix USA
25Bloodworks Majjistral, Seattle, WA
26WNY BloodCare, NY, l-Istati Uniti
27Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Centre, Los Angeles, CA, l-Istati Uniti
28Ċentru tal-Emofilja u t-Trombożi tal-Università ta 'Colorado, Kampus Mediku Anschutz, Aurora, CO
29Ċentru Mediku tal-Università Vanderbilt, Nashville, TN, USA
30uniQure BV, Amsterdam, l-Olanda / uniQure Inc. Lexington, MA, l-Istati Uniti
31Sptar Universitarju ta ’Frankfurt, Frankfurt, il-Ġermanja
Steven W Pipe, MD1, Michael Recht, MD, PhD2, Nigel S Key, MBChB3, Frank WG Leebeek, MD4, Giancarlo Castaman, MD5, Susan U Lattimore, RN6, Paul van der Valk, MD6, Kathelijne Peerlinck, MD, PhD7, Michael Coppens, MD, PhD8, Niamh O'Connell, MB PhD9, K. John Pasi, MBChB, PhD10, Peter Kampmann, MD11, Karina Meijer, MD, PhD.12, Annette von Drygalski, MD, PharmD13, Guy Young, MD14, Cedric Hermans, MD15, Jan Astermark, MD, PhD16, Robert Klamroth, MD, PhD17, Richard S Lemons, MD, PhD18, Nathan Visweshwar, MD19, Shelley Crary, MD20, Rashid Kazmi, MBBS21, Emily Symington, MBChB22, Miguel A Escobar, MD23, Esteban Gomez, MD24, Rebecca Kruse-Jarres, MD, MPH25, Adam Kotowski, MD26, Doris Quon, MD, PhD27, Michael Wang, MD28, Allison P Wheeler, MD29, Eileen K Sawyer, PhD30, Stephanie Verweij30, Valerie Colletta, MSc30, Naghmana Bajwa, MD, Robert Gut, MD, PD30, Wolfgang Miesbach, MD, PhD31
1Università ta 'Michigan, Ann Arbor, MI, USA
2Oregon Health & Science University, Portland, OR, USA
3Università ta 'North Carolina, Chapel Hill, NC, USA
4Erasmus MC, Ċentru Mediku Universitarju, Rotterdam, l-Olanda
5Ċentru għal Disturbi ta ’Fsada u Koagulazzjoni, Sptar Universitarju Careggi, Firenze, l-Italja
6Universitair Medisch Centrum Utrecht, l-Olanda
7Dipartiment tal-Mediċina Vaskulari u l-Emostasi u ċ-Ċentru tal-Emofilja, Sptarijiet Universitarji Leuven, Leuven, il-Belġju
8Dipartiment tal-Mediċina Vaskulari, Xjenzi Kardjovaskulari ta 'Amsterdam, Ċentri Mediċi ta' l-Università ta 'Amsterdam, Università ta' Amsterdam, Amsterdam, l-Olanda
9Iċ-Ċentru Nazzjonali tal-Koagulazzjoni, l-Isptar St James, Dublin, l-Irlanda
10Royal London Haemophilia Centre, Barts u l-London School of Medicine and Dentistry, Londra, ir-Renju Unit
11Rigshospitalet, Kopenħagen, id-Danimarka
12Ċentru Mediku Universitarju Groningen, Groningen, l-Olanda
13Università ta 'California San Diego, La Jolla, CA, USA
14L-Isptar tat-Tfal Los Angeles, l-Iskola tal-Mediċina Keck ta 'l-Università ta' Southern California, Los Angeles, CA, l-Istati Uniti
15Cliniques Universitaires Saint ‐ Luc, Université Catholique de Louvain, Brussell, il-Belġju
16Sptar Universitarju Skåne, Malmö
17Vivantes Klinikum im Friedrichshain, Berlin, il-Ġermanja
18Università ta 'Utah, Salt Lake City, UT, USA
19Università ta 'South Florida, Tampa, FL, USA
20Università ta 'Arkansas għax-Xjenzi Mediċi, AR, USA
21Università ta 'l-Isptar Southampton NHS Foundation Trust; Southampton, ir-Renju Unit
22Università ta 'Cambridge - Addenbrooke's Hospital, Cambridge, ir-Renju Unit
23Ċentru tax-Xjenza tas-Saħħa tal-Università ta 'Texas fi Houston, Houston, TX, l-Istati Uniti
24Phoenix Children Hospital, Phoenix USA
25Bloodworks Majjistral, Seattle, WA
26WNY BloodCare, NY, l-Istati Uniti
27Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Centre, Los Angeles, CA, l-Istati Uniti
28Ċentru tal-Emofilja u t-Trombożi tal-Università ta 'Colorado, Kampus Mediku Anschutz, Aurora, CO
29Ċentru Mediku tal-Università Vanderbilt, Nashville, TN, USA
30uniQure BV, Amsterdam, l-Olanda / uniQure Inc. Lexington, MA, l-Istati Uniti
31Sptar Universitarju ta ’Frankfurt, Frankfurt, il-Ġermanja