Note: This story was updated Jan. 20, 2026, to correct data on the number of boys on preventive FIX replacement therapy at the time of the analysis. Using longer-acting factor IX (FIX) products — known as extended half-life (EHL) products, or EHL-FIX — safely reduced factor use and helped children…
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People with hemophilia A and their caregivers, as well as healthcare providers, have shown a strong interest in a new blood-testing device being developed to make blood clotting tests faster and easier to access, according to a recent study. The device, called EnzySystem, allows for measuring the activity of…
A new study highlights a hidden emotional burden for families living with hemophilia: the deep sense of guilt felt by parents, particularly mothers, who pass the condition on to their children. However, researchers found that this weight can be significantly lifted through better access to medical information, professional counseling,…
After five years of follow-up, Hemgenix (etranacogene dezaparvovec-drlb) was associated with durable factor IX (FIX) levels, lower bleeding rates, and a substantial reduction in the need for regular FIX infusions, with a generally favorable long-term safety profile in men with severe or moderately severe hemophilia B. These data…
In 2025, Hemophilia News Today delivered readers timely reporting on research, therapeutic options, and clinical trials for hemophilia. Below is a list of the top five most-read stories we published this year. As we enter 2026, we look forward to continuing to support and serve as a source…
BBM-H901 (dalnacogene ponparvovec), a gene therapy that’s approved in China for adults with moderate to severe hemophilia B, was shown to significantly reduce bleeding rates among hemophilia B patients in clinical trials, a new study shows. Hemophilia B is a genetic disorder caused by mutations in the gene that…
Hympavzi (marstacimab) reduced bleeding rates and improved quality of life in adults and adolescents with hemophilia A or B who have inhibitors (antibodies against standard replacement therapies). That’s according to final data from the Phase 3 BASIS study (NCT03938792), which demonstrated the treatment met its main goal of…
A newborn in China was diagnosed with hemophilia B after a ruptured liver caused significant abdominal bleeding, according to a case report. Due to early detection, timely surgical repair, and immediate factor-replacement therapy, the infant made a full recovery. This case shows that “neonatal hepatic rupture may be…
A 32-year-old man was diagnosed with hemophilia A after a minor injury caused disproportionate right eye bleeding, with a buildup of pressure inside the eye and vision loss, a new U.S. case study reports. According to the researchers, this case demonstrates that an eye bleed can be a rare…
Beqvez (fidanacogene elaparvovec-dzkt), a discontinued gene therapy for hemophilia B, was shown to help reduce joint bleeding in Phase 3 clinical testing. That’s according to new analysis results from an ongoing global study that assessed the treatment’s effectiveness and found positive results. Specifically, “through 15 months, 12 of…
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