The genetic blood disorder hemophilia B, caused by factor IX (FIX) deficiency, can be effectively controlled with gene replacement therapies, particularly when used for prevention. But because barriers to prophylaxis that include intravenous use, frequent dosing and medical costs are many, new research for better therapies is emerging. Researchers at the University of Wisconsin School of…
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New therapies for hemophilia B, a genetic blood disorder caused by factor IX (FIX) deficiency, are emerging due to barriers with current prophylaxis. These include gene replacement therapies and novel recombinant FIX fusion proteins that increase dosing intervals. For hemophilia A, embryonic stem cell transplants show promise, and some patients with transient inhibitors to Factor VIII may delay therapy. Catalyst Biosciences is also preparing a Phase 3 trial for MarzAA to treat bleeding episodes in hemophilia A or B patients with inhibitors.
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Patient treatment for hemophilia B has come a long way since the introduction of factor IX (FIX) concentrates. In a recent commentary published in the journal Blood and titled “A new era for hemophilia B treatment,” researchers from Oregon Health and Science University and Bloodworks Northwest highlighted recent advances in…
Hemophilia A patients who receive replacement therapies of factor VIII often develop inhibitors against factor concentrates which hinders the effectiveness of the therapy, increases disability, and reduces quality of life. Although immune tolerance induction (ITI) can reduce inhibitors against factor VIII concentrates, the study “Long-term course of…
After receiving guidance from both the U.S. Food and Drug Administration and the European Medicines Agency, Catalyst Biosciences is preparing to launch a pivotal Phase 3 clinical trial assessing marzeptacog alfa activated (MarzAA) for the treatment of bleeding episodes in people hemophilia A or B with…
Transplants of embryonic stem (ES) cells that secrete the human factor VIII (FVIII) may be a promising therapeutic approach for patients with hemophilia A, according to an animal study conducted by researchers at the Nara Medical University School of Medicine in Japan. The study, “Therapeutic approaches for treating hemophilia…
Facial and scalp swelling can be indicators of childhood hemophilia, but for children who are misdiagnosed and treated incorrectly, the effects of the disease’s elevated bleeding predisposition can be catastrophic. In the study, “Facial and Scalp Swelling in the Pediatric Population With Hemophilia: A Diagnosis Pitfall,” published in…
Blood levels of two proteins — C-reactive protein (CRP) and vascular endothelial growth factor (VEGF) — involved in inflammation and blood vessel formation are significantly associated with, and may help predict, acute bleeding in the joints of people with severe hemophilia A, a study has found. The study,…
Hemophilia can affect many aspects of life, including sexual health. If you have hemophilia, sexual activity is generally safe as long as certain precautions are taken.
Hemophilia A is a rare genetic disorder that affects the blood’s ability to clot properly. It is the most common form of hemophilia, responsible for 80% of all cases.
Hemophilia is a rare disorder in which a person’s blood is not able to clot properly, resulting in unusually easy and prolonged bleeding.