In boys and men with severe hemophilia A, joint damage accumulates with age, and can be prevented if replacement therapy is started at a younger age, according to a small, single-center study in China. In addition, poor adherence to preventive, or prophylactic treatment was significantly associated with greater…

Administration of once-weekly Hemlibra (emicizumab-kxwh) significantly reduced bleeding rates in patients with Hemophilia A compared to other clotting factor VIII prophylaxis (preventive) therapy, according to the latest results of the Phase 3 HAVEN 3 study announced by Roche. Developed by Chugai Pharmaceutical, Roche, and Genentech,…

A gene therapy designed to provide hemophilia A patients with a highly functional version of factor VIII (FVIII), the blood clotting protein they lack, could offer more sustained bleed control than existing gene therapies, preclinical research suggests. The current approach in hemophilia A gene therapy is to provide a…

BAY 94‐9027, an investigational factor VIII replacement therapy being developed by Bayer for severe hemophilia A, is able to stay longer in patients’ blood than other standard therapies, according to a study analyzing data from three…

A new study highlights a hidden emotional burden for families living with hemophilia: the deep sense of guilt felt by parents, particularly mothers, who pass the condition on to their children. However, researchers found that this weight can be significantly lifted through better access to medical information, professional counseling,…

The first patient in a Phase 1/2 clinical trial (“the Alta trial”) designed to assess safety and tolerability of SB-525 gene therapy has received treatment. Sangamo Therapeutics, in a collaboration with Pfizer, is developing  the SB-525 gene therapy to correct the Factor VIII (FVIII) defect in hemophilia A…

Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up to two years in people with hemophilia A, a long-term extension study shows. Findings were reported in the study “BAY 81-8973 demonstrated efficacy, safety and joint status improvement…

Half of the children in Chile with severe hemophilia A and B exhibit joint damage despite receiving preventive treatment with clotting factors, a study reports. Damage to the cartilage is the main sign of joint impairment in children older than 8 years, while synovitis, or inflammation of the synovial…

Health Canada has approved Altuviiio (efanesoctocog alfa) to treat children, adolescents, and adults with hemophilia A, with the aim of preventing and controlling bleeds, including those occurring around the time of surgery. “This approval represents important progress in the treatment of patients with hemophilia A in Canada,” Stephanie…

Hemophilia is sometimes a surprise in families. Research shows that approximately 30% of cases of hemophilia A and B are sporadic, meaning there’s no known family history. “This happens when a new alteration in the F8 or F9 gene occurs by chance in an egg cell or…