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Data from two Phase 3 studies demonstrating fitusiran ...
Fitusiran is a novel subcutaneous siRNA prophylactic investigational therapy designed to lower antithrombin levels with the goal of enhancing thrombin generation to rebalance hemostasis in people with hemophilia, regardless of type or inhibitor status.
Efficacy and safety of fitusiran prophylaxis in people with ...
We evaluated the efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors. Methods: This multicentre, randomised, open-label phase 3 study was done at 26 sites (primarily secondary or tertiary centres) in 12 countries.
Antithrombin lowering in hemophilia: a closer look at fitusiran
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 in haemophilia: a breakthrough drug with many ...
One of the rebalancing drugs, fitusiran, an investigational small interfering RNA agent, reduces antithrombin synthesis in the hepatocytes, ultimately rebalancing haemostasis. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia.
Fitusiran prophylaxis in people with severe haemophilia A or ...
Fitusiran, a subcutaneous investigational siRNA therapeutic, targets antithrombin with the goal of rebalancing haemostasis in people with haemophilia A or haemophilia B, regardless of inhibitor status. We aimed to evaluate the efficacy and safety of fitusiran prophylaxis in people with severe haemophilia without inhibitors. Methods.
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