An experimental prenatal cell and gene therapy designed to restore production of the missing clotting protein in hemophilia A showed maternal safety signs in a U.S. study in sheep. The therapy, called PLC-mcoET3, is designed to transplant genetically engineered cells into the fetus so they can produce factor VIII…
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At the World Health Assembly (WHA), member states of the World Health Organization (WHO) unanimously adopted a resolution on Global Action to Advance Health Equity for People with Hemophilia and Other Bleeding Disorders to support better diagnosis, treatment, and fairer care for people with hemophilia and other bleeding…
People with hemophilia still experience significant unmet health needs despite receiving preventive treatment, according to an international study. “Despite current treatment, many [people with hemophilia] continue to experience limitations to their daily activities, productivity, and physical independence,” researchers wrote. Specific problems that emerged in the survey included breakthrough…
Hemlibra (emicizumab) alongside immunosuppressive therapy appears safe for use in acquired hemophilia A (AHA), according to initial results of a real-world study in Japan, the only country where Hemlibra has approval for this indication. “At this interim analysis, there were no unexpected safety concerns for … patients with…
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…
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…
