Haemophilia A and B are rare congenital, recessive X-linked disorders caused by lack or
deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease …

The success story of hemophilia care first began in the 1970s, when the availability of
plasma-derived concentrates of coagulation factor VIII (FVIII) and factor IX (FIX) provided …

Background Valoctocogene roxaparvovec delivers a B-domain–deleted factor VIII coding
sequence with an adeno-associated virus vector to prevent bleeding in persons with severe …

Prophylaxis with emicizumab, a subcutaneously administered bispecific humanized
monoclonal antibody, promotes effective hemostasis in persons with hemophilia A (PwHAs) …

Background Fitusiran, a subcutaneous investigational small interfering RNA therapeutic,
targets antithrombin to rebalance haemostasis in people with haemophilia A or haemophilia …

With the goal of emphasizing the striking advances that materialized in hemophilia care
particularly in the last 10 years, the progress of knowledge that started from the 1970s will …

Congenital haemophilia A (factor VIII deficiency) and B (factor IX deficiency) are X-linked
bleeding disorders. Replacement therapy has been the cornerstone of the management of …

Emicizumab, a bispecific humanized monoclonal antibody, bridges activated factor IX (FIX)
and FX to restore the function of missing activated FVIII in hemophilia A. Emicizumab …

Acquired hemophilia A (AHA) is a severe bleeding disorder caused by inhibiting
autoantibodies to coagulation factor VIII (FVIII). For hemostatic treatment, bypassing agents …

Background Clinical trial data are scarce for the use of prophylaxis in people with non-
severe haemophilia A. The HAVEN 6 study aims to assess safety and efficacy of …