Children with severe hemophilia B who develop antibodies, also known as inhibitors, against coagulation factor IX (FIX) replacement therapies continue to face major treatment challenges, according to a small study in the U.K. The study found that attempts to eliminate these inhibitors using an approach called immune tolerance induction…
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With a new label expansion cleared by the U.S. Food and Drug Administration (FDA), Hympavzi (marstacimab-hncq) is now approved for preventing or reducing the frequency of bleeds in all people with hemophilia A or hemophilia B, ages 6 and older, regardless of inhibitor status. The injectable therapy…
A proposed bill in the U.S. House of Representatives — one dubbed the FED UP Act for short — is aiming to reduce barriers to care for women and girls with bleeding disorders, including those who carry hemophilia-related genes. The legislation, fully, the Fostering Effective Diagnosis and Treatment…
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…
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…
