In the large clinical trial that supported the 2024 approval of Hympavzi (marstacimab) for adults and children with hemophilia, use of the prophylactic injection therapy was found to help prevent bleeding episodes. Breakthrough bleeds that did occur, or minor surgical procedures that were needed, were generally effectively treated…
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The European Commission has approved Hympavzi (marstacimab) to treat certain people with hemophilia A or hemophilia B who have inhibitors, the therapy’s maker, Pfizer, announced. Hympavzi was approved in Europe in 2024 to treat patients with hemophilia A or B ages 12 and older who…
People with hemophilia, particularly those with more advanced joint disease, or hemophilic arthropathy, show altered walking patterns likely linked to reduced ankle mobility, a new study in Germany has found. To cope with these gait alterations, people with hemophilia may develop compensatory strategies that could make walking less efficient…
Denecimig (Mim8) was significantly better than on-demand or standard preventive treatments at reducing bleeding episodes in people with hemophilia A, regardless of inhibitor status. That’s according to published data from the FRONTIER2 trial (NCT05053139), which evaluated the efficacy and safety of once-monthly and once-weekly denecimig treatment in adults…
The U.S. Food and Drug Administration (FDA) has granted both fast track and rare pediatric disease designations to Expression Therapeutics’ investigational stem cell therapy for hemophilia A. Each of these designations is intended to speed development of a candidate therapy. This stem cell therapy was designed as a potentially…
Many women carrying hemophilia-related mutations are not receiving the medical attention they need, according to a large-scale multinational study. Despite being treated in established healthcare systems, these women, often referred to as hemophilia carriers, frequently remain undiagnosed and excluded from official patient registries. Researchers found that a lack…
Bemiltenase alfa, an experimental factor X (FX) activator, helped with on-demand bleeding control for people with hemophilia A and B who had developed inhibitors against standard factor replacement therapies, according to results from early clinical trials. Inhibitors are antibodies that may reduce the effectiveness of hemophilia treatments, making it…
Roctavian (valoctocogene roxaparvovec-rvox) was associated with fewer bleeding episodes and less need for preventive treatment, or prophylaxis, in most adult men with severe hemophilia A for up to seven years after a single infusion, according to final results from a completed clinical trial. The Phase 1/2 clinical…
A computer-aided diagnostic tool using artificial intelligence (AI) can accurately detect joint bleeds in adults with hemophilia, a study showed. The researchers said the findings may lead to a way for patients to obtain ultrasound images at home, rather than traveling to a facility. “The CAD [computer-aided diagnosis] system…
Regular and sustained physical exercise — including aerobic activities, strength training, and balance exercises — may help ease chronic pain, improve physical performance, and enhance quality of life in people with hemophilia of all ages, according to a review study. The researchers found that exercise is generally safe when…
