While there have been advances in hemophilia B treatment, there is still desire for a new treatment option for people with moderate to severe hemophilia—one that offers people with the condition an innovative option to meet their needs.
Even people with hemophilia B who regularly receive factor IX (FIX) replacement can still experience breakthrough bleeds. When this happens, it can lead to joint damage, pain, and other long-term problems.
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Prophylactic FIX replacement requires lifelong dedication to a burdensome schedule and potential side effects. Veins can also collapse or harden, making ongoing treatment challenging.
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While even a small increase in FIX levels can dramatically reduce bleeding and its consequences, higher and more consistent factor levels would provide better protection.
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Both patients and caregivers report anxiety and depression connected to living with or helping manage the condition.
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Even people with hemophilia B who regularly receive factor IX (FIX) replacement can still experience breakthrough bleeds. When this happens, it can lead to joint damage, pain, and other long-term problems.
1 of 4
Prophylactic FIX replacement requires lifelong dedication to a burdensome schedule and potential side effects. Veins can also collapse or harden, making ongoing treatment challenging.
2 of 4
While even a small increase in FIX levels can dramatically reduce bleeding and its consequences, higher and more consistent factor levels would provide better protection.
3 of 4
Both patients and caregivers report anxiety and depression connected to living with or helping manage the condition.
4 of 4
Dr. George discusses how the needs of people with hemophilia B might be addressed by gene therapy
Be one of the first to hear news of advances in gene therapy for hemophilia B.
