Højdepunkter fra det 62. ASH årsmøde og udstilling
Første data fra fase 3 HOPE-B-genterapiforsøg: Effektivitet og sikkerhed af Etranacogen Dezaparvovec (AAV5-Padua hFIX-variant; AMT-061) hos voksne med svær eller moderat-svær hæmofili B behandlet uanset præ-eksisterende antikapsidneutralisering Antistoffer
Steven W. Pipe, MD1, Michael Recht, MD, ph.d.2, Nigel S. Key, MD3, Frank WG Leebeek, MD, ph.d.4, Giancarlo Castaman, MD5, Susan U. Lattimore, RN6, Paul Van Der Valk7,8,9, Kathelijne Peerlinck, MD, ph.d.10, Michiel Coppens, MD11, Niamh O'Connell, MD, ph.d.12, John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH13, Peter Kampmann, MD14, Karina Meijer, MD, ph.d.15, Annette von Drygalski, MD, PharmD16, Guy Young, læge17, Cedric Hermans, MD, MRCP, PhD18, Jan Astermark, MD, ph.d.19,20, Robert Klamroth, MD, ph.d.21, 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, MD29Adam Kotowski, MD30, Doris Quon, MD, ph.d.31, Michael Wang, MD32, Allison P. Wheeler, MD33, Eileen K Sawyer, ph.d.34, Stephanie Verweij, BSc35, Valerie Colletta, cand36, Naghmana Bajma, MD37, Robert Gut, MD, ph.d.36 og Wolfgang A. Miesbach, MD, ph.d.38
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, Holland
5Azienda Ospedaliera Universitaria Careggi, Firenze, Italien
6Oregon Health & Science University, Portland
7Vrije Universiteit Medical Center, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Holland
9Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Holland
10Institut for Vaskulær Medicin og Hæmostase og Hæmofili Center, Universitetshospitaler Leuven, Leuven, Belgien
11Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Holland
12Det nationale koagulationscenter, St James Hospital, Dublin, Irland
13Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, Storbritannien
14Rigshospitalet, København, Danmark
15University Medical Center Groningen, Groningen, Holland
16University of California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, børnehospital Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Bruxelles, Belgien
19Institut for Translationsmedicin-Klinisk Koagulation, Lund Universitet, Malmø, Sverige
20Skåne Universitetshospital, Malmø, 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 børnehospital, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Hemophilia Center i det vestlige 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 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
38Universitetshospital Frankfurt, Frankfurt am Main, Tyskland
Steven W. Pipe, MD1, Michael Recht, MD, ph.d.2, Nigel S. Key, MD3, Frank WG Leebeek, MD, ph.d.4, Giancarlo Castaman, MD5, Susan U. Lattimore, RN6, Paul Van Der Valk7,8,9, Kathelijne Peerlinck, MD, ph.d.10, Michiel Coppens, MD11, Niamh O'Connell, MD, ph.d.12, John Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH13, Peter Kampmann, MD14, Karina Meijer, MD, ph.d.15, Annette von Drygalski, MD, PharmD16, Guy Young, læge17, Cedric Hermans, MD, MRCP, PhD18, Jan Astermark, MD, ph.d.19,20, Robert Klamroth, MD, ph.d.21, 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, MD29Adam Kotowski, MD30, Doris Quon, MD, ph.d.31, Michael Wang, MD32, Allison P. Wheeler, MD33, Eileen K Sawyer, ph.d.34, Stephanie Verweij, BSc35, Valerie Colletta, cand36, Naghmana Bajma, MD37, Robert Gut, MD, ph.d.36 og Wolfgang A. Miesbach, MD, ph.d.38
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, Holland
5Azienda Ospedaliera Universitaria Careggi, Firenze, Italien
6Oregon Health & Science University, Portland
7Vrije Universiteit Medical Center, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Holland
9Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Holland
10Institut for Vaskulær Medicin og Hæmostase og Hæmofili Center, Universitetshospitaler Leuven, Leuven, Belgien
11Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Holland
12Det nationale koagulationscenter, St James Hospital, Dublin, Irland
13Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, Storbritannien
14Rigshospitalet, København, Danmark
15University Medical Center Groningen, Groningen, Holland
16University of California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, børnehospital Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Bruxelles, Belgien
19Institut for Translationsmedicin-Klinisk Koagulation, Lund Universitet, Malmø, Sverige
20Skåne Universitetshospital, Malmø, 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 børnehospital, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Hemophilia Center i det vestlige 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 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
38Universitetshospital Frankfurt, Frankfurt am Main, Tyskland