Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Hemophilia A and B are rare X-linked bleeding disorders caused by mutations in the genes
encoding coagulation factor VIII (FVIII) and factor IX (FIX). Hemophilia A (HA) is more …

Background Moderate-to-severe hemophilia B is treated with lifelong, continuous
coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims …

This new edition of the World Federation of Hemophilia (WFH) guidelines for the
management of hemophilia comes at an exciting time in the evolution of the diagnosis and …

Background Once-weekly efanesoctocog alfa provides high sustained factor VIII activity with
superior bleeding prevention as compared with prestudy factor VIII prophylaxis in previously …

Background Adeno-associated virus (AAV)–mediated gene therapy is under investigation as
a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years …

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

The management of perioperative bleeding involves multiple assessments and strategies to
ensure appropriate patient care. Initially, it is important to identify those patients with an …

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