When used long term, Novoeight (turoctocog alfa) safely and effectively prevented and treated bleeding episodes in people with hemophilia A in Japan, according to a real-world study. In fact, Novoeight successfully treated 91.4% of the reported bleeds, and kept bleeding under control in 85.7% of surgical procedures. Moreover,…

Clinical data from the ongoing Phase 2/3 trial data evaluating MarzAA (marzeptacog alfa activated) in patients with Hemophilia A or B with inhibitors continues to demonstrate the treatment’s potential to prevent bleedings. The most recent trial results were discussed at the 12th Annual Congress of the European Association for Haemophilia…

A first patient has been dosed in a Phase 1/2a clinical trial that’s assessing the safety and preliminary efficacy of ASC618, a second-generation gene therapy for hemophilia A. The Phase1/2a study (NCT04676048), which has been in the works since 2021, aims to enroll about 12 men with severe…

In rare instances, people with hemophilia can grow bones outside their skeletal tissue — with bone growth found in muscles and soft tissues — which significantly limit their range of motion, according to a recent case report. Surgery can improve the functional outcomes of patients with this abnormal bone…

Researchers reviewed the use of high-purity concentrates containing two plasma molecules that are lacking in patients with Von Willebrand disease (VWD), a hemorrhagic disorder. These products may be a good strategy for managing pediatric patients with VWD and hemophilia A (HA) who do not respond to other treatments. The study, “Human Von…

Treatment with marstacimab (PF-06741086), an experimental antibody-based therapy developed by Pfizer, reduced the number of bleeds in patients with severe hemophilia who took part in a Phase 1b/2 clinical study. This held true regardless of whether patients had hemophilia A or hemophilia B, or had inhibitors…

GenScript ProBio and Neoletix Biotechnology have entered into an agreement for clinical and commercial production of a lab-made version of coagulation factor VIII, a product that the companies hope will make the preventive treatment for hemophilia A more accessible to patients in China. Coagulation factor VIII, or FVIII,…

In the early days of a diagnosis, emotions run wild. Anxiety, helplessness, and anger are a few feelings that cloud the mind, and it’s difficult to even begin to understand what lies ahead. In 1996, my first son, Julian, was born and diagnosed with severe hemophilia A. In…

Gene therapy delivering the blood clotting factor VIII (FVIII) — whose lack causes hemophilia A — into the joints did better at protecting against hemophilic arthropathy (joint damage) than did administration into the bloodstream, a study in mice suggests. Its findings support the potential use of FVIII injected directly into…

The efficacy and safety profiles of preventive treatment with Hemlibra (emicizumab-KXWH) are not affected by age or the presence of some simultaneous health conditions, that is, comorbidities, in older people with hemophilia A. The findings are the result of a post hoc analysis of pooled data from Phase…