Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B

Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B

Abstract: Fitusiran is an investigational small interfering RNA therapeutic that targets antithrombin (AT) to rebalance hemostasis in people with hemophilia. Here, we present the results of a completed phase 2 open-label extension study, which evaluated the long-term safety and efficacy of fitusiran...

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Main Authors: Steven W. Pipe, Toshko Lissitchkov, Pencho Georgiev, Sarah Mangles, Inga Hegemann, Alice Trinchero, Pratima Chowdary, Adam Forbes, Liqi Feng, Laurel A. Menapace, Salim Kichou, Shauna Andersson, Marek Demissie, Margaret V. Ragni
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952924007110
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