1. Hemophilia B is a bleeding disorder people are born with. It’s genetic. We inherit genes from our parents and genes are uniquely coded instructions that tell a person’s cells what to do. They determine many things about how we look and how our bodies function.
2. A difference, or mutation, with the Factor IX gene is associated with Hemophilia B. Someone who has Hemophilia B doesn’t have enough clotting Factor IX (9). Factor IX helps the body stop bleeding. It’s called Hemophilia B because there’s another more common type of bleeding disorder called Hemophilia A. Those patients are missing Factor VIII (8).
3. The liver makes clotting factors, proteins that circulate in the body until needed. When the body is cut or injured, clotting factor contributes to the “coagulation cascade.” That’s a medical term for how the body stops bleeding – a first step in healing after an injury.
4. For a long time, people who had hemophilia died young because effective treatments didn’t exist. That dramatically changed with factor replacement therapy. People could receive, by intravenous (IV) infusion, the factor that they were missing. Today many people with hemophilia have access to treatment that enables them to enjoy normal life spans and even play some sports – with their doctor’s OK and always with recommended safety gear.
5. Gene therapy opens a new avenue of potential treatments for Hemophilia B. Can a treatment go to the source, and alter the mutation in the F9 gene? Many questions remain. But together, researchers and patients in clinical trials are working hard and dreaming big as they seek out potential game-changing treatments.