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Antithrombin Lowering in Hemophilia: a Closer Look at Fitusiran by Guy Young; Peter J. Lenting; Stacy E. Croteau; Beatrice Nolan; Alok Srivastava is a Medicine article available to read on EtoBox.
What is Antithrombin Lowering in Hemophilia: a Closer Look at Fitusiran about?
Thrombin is a key enzyme in the maintenance of normal hemostatic function and is the central product of an interconnected set of simultaneously occurring cellular and proteolytic events. Antithrombin (AT) is a natural anticoagulant that downregulates different components of the clotting process, particularly thrombin generation. In good health, well-regulated hemostasis is the result of a balance between procoagulant and anticoagulant elements. Cumulative understanding of the regulation of thrombin generation and its central role in hemostasis and bleeding disorders has led to the clinical development of therapeutic strategies that aim to rebalance hemostasis in individuals with hemophilia and other coagulation factor deficiencies to improve bleeding phenotype. The aim of this review is to discuss the rationale for AT lowering in individuals with hemophilia, with a focus on fitusiran, its mechanism of action, and its potential as a prophylactic therapy for individuals with hemophilia A or B, with or without inhibitors. Fitusiran is an investigational small, interfering RNA therapeutic that targets and lowers AT. It is currently in phase III clinical trials and results have shown it
Who reads Antithrombin Lowering in Hemophilia: a Closer Look at Fitusiran?
It is typically read by researchers, students, and practitioners in Medicine.
- Author
- Guy Young; Peter J. Lenting; Stacy E. Croteau; Beatrice Nolan; Alok Srivastava
- Publisher
- Elsevier BV
- Published
- 2023
- Language
- EN
- Field
- Medicine (Health Sciences)