The severity levels of hemophilia are determined by the level of residual/remaining clotting factor (factor VIII or factor IX) activity in the patient’s blood, which is denoted by the percentage of factor activity in the blood (or international units per ml of blood – IU/ml). According to the World Federation of Hemophilia,…
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The Most Commonly Asked Questions About Hemophilia
Hemophilia is it a genetic disorder where the body doesn’t produce clotting factor, which means blood can’t clot. This makes patients susceptible to both heavy external and internal bleeding, which if left untreated, can be life-threatening. Thanks to Hemophilia Village, we have the answers to some of the most commonly…
Scientists report that hemophilia is three times more prevalent in men worldwide than previously thought and still associated with a poorer life expectancy, especially in low-income countries. Findings of the study, “Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries,”…
Novo Nordisk has entered into a collaboration agreement with Bluebird Bio to develop new therapies based on gene editing for a series of genetic disorders, including hemophilia. The companies have agreed to work together on a three-year research project that will focus on the identification and…
The protein missing or defective in people with hemophilia A, called blood coagulation factor VIII (FVIII), is able on its own to stimulate immune responses in a mouse model, a study suggests. This immune response to FVIII is dependent on a set of distinct immune cells within the spleen,…
Updated results from the Alta trial show that a single infusion with the highest dose of SB-525, an investigational gene therapy, yields dose-dependent and durable increases in clotting factor VIII (FVIII). The trial, in adults with severe hemophilia A , found no bleeding episodes up…
Treatment of severe hemophilia A with Eloctate and hemophilia B with Alprolix was safe and improved the patients’ annualized bleed rates (ABRs) over four years, according to the results of two extension studies announced by Bioverativ and Sobi. The two open-label extension studies — ASPIRE (NCT01454739), and B-YOND (NCT01425723) — evaluated…
Sevenfact (eptacog beta) is a bypassing agent that’s approved to treat bleeding episodes in people with hemophilia A or hemophilia B who developed inhibitors, or neutralizing antibodies, against clotting factor VIII (FVIII) or factor IX (FIX).