A single infusion of Beqvez (fidanacogene elaparvovec-dzkt), a now-discontinued gene therapy for hemophilia B, led to sustained production of factor IX (FIX) — the clotting protein missing in people with the condition — for up to six years, reducing bleeding episodes and eliminating the need for routine preventive…
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Children with hemophilia in areas with limited medical resources face a high risk of fatal brain bleeds, a danger that is dramatically worsened by delays in seeking emergency care. A new study from Thailand reveals that without access to modern preventive therapies, more than a third of pediatric patients…
An experimental therapy may help remove harmful antibodies that reduce the effectiveness of factor replacement therapies in hemophilia A, potentially offering a new way to overcome one of the most significant complications of standard care. The therapy uses chimeric autoantibody receptor T cells, or CAAR-T cells. These are…
In boys and men with severe hemophilia A, starting prophylaxis — preventive treatment given regularly to reduce bleeding — early and maintaining regular physical activity were each linked to a lower risk of hemophilic arthropathy, a progressive form of joint damage caused by repeated bleeding, according to a real-world…
A young woman who sought emergency care for severe pain and swelling of her left hand five weeks after giving birth was ultimately diagnosed with acquired hemophilia A — caught early enough that she did not require surgery. That’s according to a new report detailing the woman’s case, which,…
Note: This story was updated March 5, 2026, to correct that tranexamic acid and epsilon-aminocaproic acid prevent the breakdown of blood clots, rather than prevent blood clots. Dental treatments can be performed safely in people with hemophilia when proper medical precautions are followed, according to a review of studies…
Testing levels of thrombin — an enzyme that helps blood clot — in people with acquired hemophilia A could help doctors fine-tune care for these patients when both clot-promoting and blood-thinning medications are used. That’s the conclusion drawn by researchers in the Netherlands, who found that thrombin testing in…
The World Health Organization (WHO) executive board approved a resolution designed to improve access to diagnostic testing and treatment for people with hemophilia and other bleeding disorders. The resolution calls on WHO member states to take actions to improve access to quality care for people with bleeding disorders. It…
Pfizer said the U.S. Food and Drug Administration (FDA) will review the company’s request to expand approval of its preventive treatment Hympavzi (marstacimab) to cover all people with hemophilia A or B ages 6 and older. Hympavzi is FDA-approved to treat hemophilia A and B in patients ages…
While nearly one-third of people living with hemophilia are female, a new global survey reveals that many of these patients remain undiagnosed due to systemic healthcare barriers and social stigma. Researchers representing the World Federation of Hemophilia (WFH) are calling for a coordinated effort to improve diagnostic access…
