Fitusiran, a subcutaneous investigational small interfering RNA therapeutic, targets antithrombin to rebalance haemostasis in people with haemophilia A or haemophilia B, irrespective of inhibitor status. We evaluated the efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors.
Publication Type
Multicenter Study
Date of Publication
April 29, 2023
Journal
Lancet (London, England)
Volume/Issue
401/10386
ISSN
1474-547X