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 …

The vascular endothelium has several important functions, including hemostasis. The
homeostasis of hemostasis is based on a fine balance between procoagulant and …

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 …

Abstract Background Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-
associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII …

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 …

Background Emicizumab is a bispecific antibody that mimics the cofactor function of
activated factor (F) VIII. It prevents bleeds in patients with congenital hemophilia A …

Introduction Emicizumab is a bispecific monoclonal antibody developed to address the
unmet needs of clotting factor replacement therapy and has become the benchmark for …

Background Fitusiran, a subcutaneous investigational siRNA therapeutic, targets
antithrombin with the goal of rebalancing haemostasis in people with haemophilia A or …

Some non‐factor products that work by facilitating the coagulation pathway (emicizumab)
and blocking the anticoagulant pathway (fitusiran, concizumab and marstacimab) for …

Background Emicizumab is a subcutaneously administered humanized, bispecific,
monoclonal antibody approved for prophylaxis in people with hemophilia A. Methods …