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A new 13-year study published in The New England Journal of Medicine confirmed that adeno-associated virus (AAV) gene therapy—a technique using a harmless virus to deliver a working gene into the liver—can lead to long-lasting production of factor IX, the clotting protein missing in people with hemophilia B. This reduces bleeding episodes and the need for regular infusions.
Hemophilia B is a rare genetic bleeding disorder caused by a mutation in the F9 gene, resulting in little or no production of factor IX.
In the U.S., about 7,000 individuals have hemophilia B, according to a study of Hemophilia Treatment Center (HTC) patients from 2012 to 2018, adjusted for those not receiving care at HTCs.
Overall, an estimated 29,761 to 32,985 males have hemophilia, with 76.5% having hemophilia A and 23.5% hemophilia B, affecting all racial and ethnic groups.
However, women are underrepresented in studies, making it difficult to estimate prevalence among females accurately.
The study shared that those with severe hemophilia B have less than 1% of normal factor IX activity, often experiencing spontaneous bleeding that can cause joint...