First data from the phase 3 HOPE-B gene therapy trial: Efficacy and safety of etranacogene dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in adults with severe or moderate-severe hemophilia B treated irrespective of pre-existing anti-capsid neutralizing antibodies

Steven W. Pipe, Michael Recht, Nigel S. Key, Frank W.G. Leebeek, Giancarlo Castaman, Susan U. Lattimore, Paul van der Valk, Kathelijne Peerlinck, Michiel Coppens, Niamh O'Connell, John Pasi, Peter Kampmann, Karina Meijer, Annette von Drygalski, Guy Young, Cedric Hermans, Jan Astermark, Robert Klamroth, Richard S. Lemons, Nathan VisweshwarShelley Crary, Rashid Kazmi, Emily Symington, Miguel A. Escobar, Esteban Gomez, Rebecca Kruse-Jarres, Adam Kotowski, Doris Quon, Michael Wang, Allison P. Wheeler, Eileen K. Sawyer, Stephanie Verweij, Valerie Colletta, Naghmana Bajma, Robert Gut, Wolfgang A. Miesbach

Research output: Contribution to journalArticleAcademicpeer-review

8 Citations (Scopus)
Original languageEnglish
JournalBlood
Volume136
DOIs
Publication statusPublished - 19 Nov 2020

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