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Haemophilia : the Official Journal of the World Federation of Hemophilia|June 25, 2021
The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studiesMark W Skinner, Claude Négrier, Ido Paz-Priel, et al.
The Lancet. Haematology|April 1, 2023
Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trialAlok Srivastava, Savita Rangarajan, Kaan Kavakli, et al.
Haemophilia : the Official Journal of the World Federation of Hemophilia|October 22, 2024
A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary resultsCedric Hermans, Leonard A Valentino, Courtney D Thornburg, et al.
Arteriosclerosis, Thrombosis, and Vascular Biology|February 2, 2023
Macrophage Galactose Lectin Contributes to the Regulation of FVIII (Factor VIII) Clearance in Mice-Brief ReportSoracha E Ward, Thomas Guest, Ciara Byrne, et al.
Blood|March 7, 2025
Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE studyGuy Young, Kaan Kavakli, Robert Klamroth, et al.
Blood Advances|August 8, 2022
Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studiesRebecca Kruse-Jarres, Flora Peyvandi, Johannes Oldenburg, et al.
Blood|January 29, 2021
Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studiesMichael U Callaghan, Claude Negrier, Ido Paz-Priel, et al.
Research and Practice in Thrombosis and Haemostasis|April 16, 2025
Development of a novel gene editing lexicon for hemophilia: methodology and resultsCraig M Kessler, Leonard A Valentino, Courtney D Thornburg, et al.
Research and Practice in Thrombosis and Haemostasis|May 1, 2026
Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial resultsAndrew D Leavitt, Johnny Mahlangu, Priyanka Raheja, et al.
The New England Journal of Medicine|March 16, 2022
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia AMargareth C Ozelo, Johnny Mahlangu, K John Pasi, et al.
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