July 17, 2026
Interim data from an ongoing extension study looking at the long-term safety and efficacy of once-weekly, once-every-two-weeks, and once-monthly denecimig (Mim8) showed that the antibody therapy continued to maintain low bleeding rates in children, adolescents, and adults with hemophilia A.
June 30, 2026
A small study found that children with hemophilia A showed changes in certain immune cells and inflammatory responses compared to children without the condition. The type of preventative treatment they received appeared to shape the immune response in different ways, showing that Hemlibra produced fewer inflammatory responses than factor replacement therapies.
June 16, 2026
The National Bleeding Disorders Foundation has released standardized guidelines for screening for iron deficiency for people who have bleeding disorders, and the recommendation should help give doctors clear guidance on when to screen.
June 9, 2026
The FDA approved an expanded indication for Hympavzi (marstacimab), allowing it to be used in children ages 6-11 years with hemophilia B, with or without inhibitors. The expanded approval allows the treatment to become the first non-factor therapy available for pediatric patients in this age group.
May 19, 2026
A large clinical trial showed that the use of prophylactic injection therapy of Hympavzi (marstacimab) for adults and children helped prevent bleeding episodes for patients with both hemophilia A and B. Breakthrough bleeds that did occur, or minor surgical procedures that were needed were effectively treated with standard clotting factor replacement therapy.
May 5, 2026
The FDA has awarded both fast track and rare pediatric disease designations to a single-use stem cell therapy being developed for hemophilia A by Expression Therapeutics. The decision follows a Phase 1 clinical trial showing a single administration of the therapy led to sustained factor expression and the annual bleeding rate dropped to zero.
May 4, 2026
Researchers in Italy have found that prophylaxis with Mim8, a novel bispecific antibody designed to replicate function of activated factor VIII, was superior to both on-demand therapy and clotting factor concentrate prophylaxis in reducing annualized rate of bleeding events, regardless of inhibitor status.
April 17, 2026
A study published in Pain Management Nursing reviewed published research about the effect of physical excercise, including aerobic, strength training, and balance exercises, found that regular exercise may help ease chronic pain for those with hemophilia.
April 3, 2026
A study in Greece compared the YKL-40 protein levels - a protein associated with inflammation - in a small group of boys and men with severe hemophilia A and found that those with known joint damage had signficantly higher levels. The findings offer a potential new way to monitor joint damage in bleeding disorders patients.
March 20, 2026
A small survey in Germany found that many people with severe hemophilia are not interested in receiving gene therapy. Researchers found that despite having only a general knowledge of the treatment option, more than half of those surveyed had a predominantly rejective attitude toward the therapy.
March 18, 2026
CSL Behring announced that its hemophilia B gene therapy, Hemgenix, was experiencing a temporary global stockout of the treatment. The company shared that the supply issue was not related to safety or effectiveness of the medication, but reflects the complexity of manufacturing gene therapies while maintaining regulatory and quality standards.
March 17, 2026
A Phase 1 pilot study in China showed their experimental gene therapy GS001 safely increased the levels of factor VIII for hemophilia A patients for almost three years. The treatment reduced bleeds and the need for replacement therapy.
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