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.
Publication Type
Journal Article
Date of Publication
May 1, 2023
Journal
The Lancet. Haematology
Volume/Issue
10/5
ISSN
2352-3026