Höjdpunkter från det 62: e ASH-årsmötet och utställningen
Första data från fas 3 HOPE-B-genterapiprov: Effektivitet och säkerhet av Etranacogen Dezaparvovec (AAV5-Padua hFIX-variant; AMT-061) hos vuxna med svår eller måttligt svår hemofili B behandlad oberoende av existerande antikapsidneutralisering Antikroppar
Steven W. Pipe, MD1, Michael Recht, MD, doktor2, Nigel S. Key, MD3, Frank WG Leebeek, MD, PhD4, Giancarlo Castaman, MD5Susan U. Lattimore, RN6, Paul Van Der Valk7,8,9, Kathelijne Peerlinck, MD, doktor10Michiel Coppens, MD11, Niamh O'Connell, doktor, doktor12, John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH13, Peter Kampmann, MD14, Karina Meijer, doktor, doktor15Annette von Drygalski, MD, PharmD16, Guy Young, MD17, Cedric Hermans, MD, MRCP, PhD18, Jan Astermark, doktor, doktor19,20, Robert Klamroth, doktor, doktor21Richard 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, MD29Adam Kotowski, MD30, Doris Quon, MD, doktor31, Michael Wang, MD32, Allison P. Wheeler, MD33, Eileen K Sawyer, doktorsexamen34, Stephanie Verweij, kandidatexamen35, Valerie Colletta, civilingenjör36, Naghmana Bajma, MD37, Robert Gut, MD, doktor36 och Wolfgang A. Miesbach, MD, doktor38
1University of Michigan, Ann Arbor, MI
2Oregon Health & Science University, Portland, OR
3University of North Carolina, Chapel Hill, NC
4Erasmus MC, University Medical Center Rotterdam, Rotterdam, Nederländerna
5Azienda Ospedaliera Universitaria Careggi, Florens, Italien
6Oregon Health & Science University, Portland
7Vrije Universiteit Medical Center, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Nederländerna
9Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Nederländerna
10Institutionen för vaskulär medicin och hemostas och hemofilicenter, universitetssjukhus Leuven, Leuven, Belgien
11Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Nederländerna
12National Coagulation Center, St James Hospital, Dublin, Irland
13Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, Storbritannien
14Rigshospitalet, Köpenhamn, Danmark
15Universitetsmedicinskt centrum Groningen, Groningen, Nederländerna
16University of California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, Barnsjukhus Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Bryssel, Belgien
19Institutionen för translationell medicin-klinisk koagulation, Lunds universitet, Malmö
20Skåne universitetssjukhus, Malmö, Sverige
21Vivantes Klinikum im Friedrichshain, Berlin, Tyskland
22University of Utah, Salt Lake City, UT
23University of South Florida, Tampa, FL
24University of Arkansas for Medical Sciences, Little Rock, AR
25University Hospital Southampton NHS Foundation Trust, Southampton, Storbritannien
26Cambridge University – Addenbrooke's Hospital, Cambridge, Storbritannien
27University of Texas Health Science Center i Houston, Houston, TX
28Phoenix Children's Hospital, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Hemophilia Center i västra New York, New York
31Los Angeles ortopediska sjukhus, Orthopedic Hemophilia Treatment Center, Los Angeles, CA
32Anschutz Medical Campus, University of Colorado, School of Medicine, Hemophilia and 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
38Universitetssjukhuset Frankfurt, Frankfurt, Tyskland
Steven W. Pipe, MD1, Michael Recht, MD, doktor2, Nigel S. Key, MD3, Frank WG Leebeek, MD, PhD4, Giancarlo Castaman, MD5Susan U. Lattimore, RN6, Paul Van Der Valk7,8,9, Kathelijne Peerlinck, MD, doktor10Michiel Coppens, MD11, Niamh O'Connell, doktor, doktor12, John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH13, Peter Kampmann, MD14, Karina Meijer, doktor, doktor15Annette von Drygalski, MD, PharmD16, Guy Young, MD17, Cedric Hermans, MD, MRCP, PhD18, Jan Astermark, doktor, doktor19,20, Robert Klamroth, doktor, doktor21Richard 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, MD29Adam Kotowski, MD30, Doris Quon, MD, doktor31, Michael Wang, MD32, Allison P. Wheeler, MD33, Eileen K Sawyer, doktorsexamen34, Stephanie Verweij, kandidatexamen35, Valerie Colletta, civilingenjör36, Naghmana Bajma, MD37, Robert Gut, MD, doktor36 och Wolfgang A. Miesbach, MD, doktor38
1University of Michigan, Ann Arbor, MI
2Oregon Health & Science University, Portland, OR
3University of North Carolina, Chapel Hill, NC
4Erasmus MC, University Medical Center Rotterdam, Rotterdam, Nederländerna
5Azienda Ospedaliera Universitaria Careggi, Florens, Italien
6Oregon Health & Science University, Portland
7Vrije Universiteit Medical Center, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Nederländerna
9Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Nederländerna
10Institutionen för vaskulär medicin och hemostas och hemofilicenter, universitetssjukhus Leuven, Leuven, Belgien
11Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Nederländerna
12National Coagulation Center, St James Hospital, Dublin, Irland
13Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, Storbritannien
14Rigshospitalet, Köpenhamn, Danmark
15Universitetsmedicinskt centrum Groningen, Groningen, Nederländerna
16University of California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, Barnsjukhus Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Bryssel, Belgien
19Institutionen för translationell medicin-klinisk koagulation, Lunds universitet, Malmö
20Skåne universitetssjukhus, Malmö, Sverige
21Vivantes Klinikum im Friedrichshain, Berlin, Tyskland
22University of Utah, Salt Lake City, UT
23University of South Florida, Tampa, FL
24University of Arkansas for Medical Sciences, Little Rock, AR
25University Hospital Southampton NHS Foundation Trust, Southampton, Storbritannien
26Cambridge University – Addenbrooke's Hospital, Cambridge, Storbritannien
27University of Texas Health Science Center i Houston, Houston, TX
28Phoenix Children's Hospital, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Hemophilia Center i västra New York, New York
31Los Angeles ortopediska sjukhus, Orthopedic Hemophilia Treatment Center, Los Angeles, CA
32Anschutz Medical Campus, University of Colorado, School of Medicine, Hemophilia and 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
38Universitetssjukhuset Frankfurt, Frankfurt, Tyskland