Høydepunkter fra det 62. ASH årsmøtet og utstillingen
Første data fra fase 3 HOPE-B-genterapiforsøk: Effekt og sikkerhet av Etranakogen Dezaparvovec (AAV5-Padua hFIX-variant; AMT-061) hos voksne med alvorlig eller moderat-alvorlig hemofili B behandlet uavhengig av eksisterende antikapsid-nøytralisering Antistoffer
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, 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 Young, 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 og Wolfgang A. Miesbach, MD, PhD38
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, Nederland
5Azienda Ospedaliera Universitaria Careggi, Firenze, Italia
6Oregon Health & Science University, Portland
7Vrije Universiteit Medical Center, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Nederland
9Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Nederland
10Institutt for vaskulær medisin og hemostase og hemofili senter, universitetssykehus Leuven, Leuven, Belgia
11Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Nederland
12The National Coagulation Center, St James Hospital, Dublin, Irland
13Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, Storbritannia
14Rigshospitalet, København, Danmark
15Universitetsmedisinske senter Groningen, Groningen, Nederland
16University of California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, Barnesykehus Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Brussel, Belgia
19Institutt for translasjonell medisin-klinisk koagulasjon, Lunds universitet, Malmö, Sverige
20Skåne universitetssykehus, 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 medisinske vitenskaper, Little Rock, AR
25Universitetssykehus Southampton NHS Foundation Trust, Southampton, Storbritannia
26Cambridge University – Addenbrooke's Hospital, Cambridge, Storbritannia
27University of Texas Health Science Center i Houston, Houston, TX
28Phoenix Children's Hospital, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Hemophilia Center of Western New York, New York
31Los Angeles ortopediske sykehus, 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
38Universitetssykehuset Frankfurt, Frankfurt, Tyskland
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, 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 Young, 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 og Wolfgang A. Miesbach, MD, PhD38
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, Nederland
5Azienda Ospedaliera Universitaria Careggi, Firenze, Italia
6Oregon Health & Science University, Portland
7Vrije Universiteit Medical Center, Amsterdam, NLD
8Universitair Medisch Centrum Utrecht, Utrecht, Nederland
9Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Nederland
10Institutt for vaskulær medisin og hemostase og hemofili senter, universitetssykehus Leuven, Leuven, Belgia
11Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Nederland
12The National Coagulation Center, St James Hospital, Dublin, Irland
13Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, Storbritannia
14Rigshospitalet, København, Danmark
15Universitetsmedisinske senter Groningen, Groningen, Nederland
16University of California San Diego, La Jolla, CA
17University of Southern California Keck School of Medicine, Barnesykehus Los Angeles, Los Angeles, CA
18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Brussel, Belgia
19Institutt for translasjonell medisin-klinisk koagulasjon, Lunds universitet, Malmö, Sverige
20Skåne universitetssykehus, 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 medisinske vitenskaper, Little Rock, AR
25Universitetssykehus Southampton NHS Foundation Trust, Southampton, Storbritannia
26Cambridge University – Addenbrooke's Hospital, Cambridge, Storbritannia
27University of Texas Health Science Center i Houston, Houston, TX
28Phoenix Children's Hospital, Phoenix, AZ
29Bloodworks Northwest, Seattle, WA
30Hemophilia Center of Western New York, New York
31Los Angeles ortopediske sykehus, 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
38Universitetssykehuset Frankfurt, Frankfurt, Tyskland