The Haemophilia Joint Health Score (HJHS), a standard tool used to assess joint health in people with bleeding disorders, including hemophilia, may underestimate joint damage, or haemarthropathy, in women and girls, a study suggests. That’s because the HJHS assesses joint mobility by comparing a person’s joint range of motion…
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A new type of gene-editing treatment being developed by researchers in Japan corrected mutations causing severe hemophilia B in mice, restoring production of the missing clotting factor IX (FIX), which causes the rare bleeding disorder. The scientists say their novel nonviral treatment — which does not use an inactivated…
In a small study, children with hemophilia A had distinct changes in certain immune cells and inflammatory signaling molecules compared with healthy children, and the type of preventive treatment they receive may shape those immune responses in different ways, according to the researchers. In lab experiments, macrophages — immune…
Nearly one-third of U.S. hemophilia treatment centers (HTCs) do not involve genetic counselors in patient care or refer patients for genetic counseling, despite widespread recognition among healthcare providers that these specialists offer valuable expertise, a U.S. survey found. Healthcare providers across treatment centers recognized the value of genetic counselors…
Researchers spent years searching for a new treatment option for people with hemophilia A or B before arriving at the therapy that became Hympavzi (marstacimab). Scientists at Pfizer, the company that markets Hympavzi, described the search in a study, “Discovery and optimization of marstacimab, a human monoclonal antibody…
Repeated dosing of the long-lasting factor replacement therapy Altuviiio (efanesoctocog alfa) over a short timeframe may not increase the risk of dangerous clotting events, according to studies in a mouse model of hemophilia A. Blood analysis showed an increase in global coagulation function, which measures the formation and…
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…
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…
