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Blood Advances|December 6, 2024
Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or BSteven W Pipe, Toshko Lissitchkov, Pencho Georgiev, et al.
Molecular Therapy. Methods & Clinical Development|September 19, 2025
Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 studyRobert Klamroth, Michael Recht, Nigel S Key, et al.
The New England Journal of Medicine|December 8, 2025
Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia BSteven W Pipe, Wolfgang Miesbach, Michael Recht, et al.
Research and Practice in Thrombosis and Haemostasis|February 3, 2025
Adeno-associated virus-based gene therapy for hemophilia-addressing the gapsWolfgang Miesbach, Paul Batty, Pratima Chowdary, et al.
Blood Advances|April 6, 2025
Completion of Phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 yearsAnnette von Drygalski, Esteban Gomez, Adam Giermasz, et al.
Journal of Thrombosis and Haemostasis : JTH|January 11, 2025
Accurate evaluation of factor VIII activity of efanesoctocog alfa in the presence of emicizumabChristophe Nougier, Steven W Pipe, Ingrid Pabinger, et al.
Blood|September 21, 2010
In vivo efficacy of platelet-delivered, high specific activity factor VIII variantsTeshell K Greene, Cheng Wang, Jessica D Hirsch, et al.
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