An investigational factor VIIa (FVIIa) therapy called GEN-0828 may be able to minimize blood loss and accelerate blood clotting at lower doses than NovoSeven, an approved medication used to control bleeding in hemophilia patients, according to a study in a mouse model of hemophilia B. Still, both treatments…
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Instead of Powerball, I have won the rare bleeding disorder lottery. I have von Willebrand disease and hemophilia B, a less common form of hemophilia that arises when a blood-clotting protein called factor IX is missing or deficient. My diagnoses are rare within a rare community. I often feel like a…
About hemophilia Hemophilia is an inherited X-linked recessive bleeding disorder, caused by a deficiency in coagulation factor VIII (hemophilia A), factor IX (hemophilia B), or factor XI (hemophilia C), that results from mutations in the clotting factor genes; it mainly affects males (hemophilia A and B, the two most common forms of…
Sangamo BioSciences announced that its zinc finger nuclease (ZFN)-mediated genome editing product, SB-FIX, has received orphan drug designation from the U.S. Food and Drug Administration (FDA), speeding its development as a potential treatment for hemophilia B. Sangamo plans to initiate a Phase 1/2 clinical trial, SB-FIX-1501, in adults with this disease later…
Chronic joint inflammation lowers the health-related quality of life in people with severe hemophilia, a European study reports, and urges physicians to be aware of this association in treating patients. The research, “The impact of severe haemophilia and the presence of target joints on health-related quality-of-life,” appeared in…
Cyklokapron (tranexamic acid) is an approved hemophilia medication that's designed to control or prevent bleeding episodes in patients, and reduce the need for replacement therapy during and following a tooth extraction.
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The Symptoms of Hemophilia
Hemophilia is a rare disorder where a person’s blood lacks a factor which enables it to clot, leading to excessive bleeding that can become life-threatening. This bleeding can occur internally or externally. According to the World Federation of Hemophilia and the National Heart, Lung…
Researchers at the University of Tokyo reviewed the status of physical activity among hemophilia patients to discuss strategies to encourage activity through a behavioral change approach by focusing on hemophilia-relevant factors such as benefits and bleeding risk, risk management of bleeding, physical activity characteristics, and difficulty in exercise adherence. The study,…
Novo Nordisk has paused three clinical trials evaluating concizumab, its experimental treatment candidate for hemophilia A and B, due to safety concerns. The three Explorer studies — the Phase 2 Explorer 5 (NCT03196297), and the global Phase 3 Explorer 7 (NCT04083781) and Explorer 8 (NCT04082429)…
Grand Life Sciences Group has entered into an exclusive collaboration agreement with Belief Biomed (BBM) to commercialize BBM-H803, BBM’s one-time investigational gene therapy for hemophilia A, in Greater China if it is eventually approved in the region. The agreement covers Mainland China, as well as Hong Kong,…