The genetic blood disorder hemophilia B, caused by factor IX (FIX) deficiency, can be effectively controlled with gene replacement therapies, particularly when used for prevention. But because barriers to prophylaxis that include intravenous use, frequent dosing and medical costs are many, new research for better therapies is emerging. Researchers at the University of Wisconsin School of…

Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively lowers bleeding rates in children and adolescents with severe hemophilia A, according to a real-life study from Israel. The therapy’s effectiveness was independent of the presence of inhibitors (antibodies) against synthetic formulations of factor VIII (FVIII) — the missing…

This past week has been a whirlwind of activity for our family. My husband, Jared, who has severe hemophilia B, experienced a knee bleed that required careful management. But that didn’t stop him from fulfilling his responsibilities around the house and making progress with his budding bottled food business.

In this video from BloodFeed – Hemophilia Bleeding Disorders Media, actor Brian Williams (who plays hematologist Dr. Young in the online comedy Stop the Bleeding) takes viewers on a tour of a hemophilia treatment center (HTC). MORE: Three tips for coping with a hemophilia…

Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively reduces bleeding rates in children younger than 12 with hemophilia type A, regardless of development of inhibitors (antibodies) against synthetic factor VIII (FVIII), data from two Phase 3 studies show. Hemlibra is a type of replacement therapy that mimics…

Efanesoctocog alfa, a long-lasting factor VIII (FVIII) replacement therapy, has now been approved in the European Union for hemophilia A. It will be marketed under the brand name Altuvoct. Altuvoct can be used as an on-demand treatment for bleeding episodes, a once-weekly prophylactic (preventive) therapy to reduce the…

Preventive therapy, or prophylaxis, in patients ages 50 and older with severe hemophilia type A  and type B was effective at reducing bleeds, including those in target joints, and improving some aspects of their quality of life. That’s according to findings from a post hoc analysis of four…

Rare Disease Day, observed this year on Feb. 29, serves as a poignant reminder of the challenges facing those who live with uncommon medical conditions. According to the event’s website, a rare disease is defined as one that affects fewer than 1 in 2,000 individuals. Among these conditions…

Fewer HIV infections and better care may explain the higher prevalence of hemophilia in male patients in Indiana between 2011 and 2013 than previously reported in six other states, according to a new study. The findings also revealed a higher utilization of Hemophilia Treatment Centers (HTCs) by this patient…

Weekly treatment with the experimental therapy marstacimab (PF-06741086) was found to significantly reduce bleed rates for adult and adolescent hemophilia A and B patients without inhibitors relative to standard therapies. In so doing, it met the main goal of the pivotal Phase 3 BASIS clinical trial, designed to…