Nearly all patients treated with a one-time gene therapy shot for hemophilia B were able to discontinue full-dose bleeding prophylaxis, says a study. Annual bleed rates fell by 64% after the lead-in period.
New approaches to treating hemophilia may soon replace the decades-old approach of using factor concentrates to control bleeding in patients with hemophilia A and hemophilia B.
Instead of taking the traditional approach of replacing what s missing, the idea of fitusiran is to short circuit the body s anticoagulation system by targeting antithrombin.
Promising execution in the clinic with programs in hemophilia B, Fabry disease and Gaucher disease; potential to deliver transformative therapies driven by.