Gene therapy directly addresses hemophilia B’s underlying cause by providing a healthy version of the F9 gene. In this way, it is expected to offer lasting bleed control with minimal or possibly no need for regular preventive therapies.
Advancing hemophilia B treatment:
The gene therapy approach
Gene therapy is a newly approved treatment option for people with hemophilia B that delivers a healthy version of the F9 gene that is mutated in people with the disease. Gene therapies have been associated with increased FIX levels, significantly reduced bleeding episodes, and a reduction or elimination of the need for routine prophylaxis. Learn more about what gene therapy is, who is eligible, and some other considerations in the articles below.
There are a number of factors that patients and their healthcare team will want to consider to determine if gene therapy is the right choice for them, including eligibility requirements, risks and benefits, and costs.
As gene therapy is relatively new treatment option for hemophilia B, it's normal to have some questions about it. Here are answers to some commonly asked questions about gene therapy for hemophilia B.
Equipping yourself with knowledge is crucial for advocating for your hemophilia B healthcare, especially if you want to ask your doctor about trying a new treatment like gene therapy.
Cazandra Campos-MacDonald, whose son lives with hemophilia, shares her experiences with hemophilia treatment centers and offers advice on how to find the right center to suit your needs.
Connecting with others affected by hemophilia B can help you find a community with whom you can share what it’s like living with hemophilia B, including talking about daily life, symptoms, and treatments.