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What you need to know about gene therapy and hemophilia B
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.
FAQs about gene therapy for hemophilia B
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.
Collaborative care: Finding the right hemophilia treatment center
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.
Building your hemophilia B community
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.
Treating iron deficiency in women with hemophilia
Iron deficiency has been a hot topic at the online hemophilia summits I’ve attended recently, and it’s encouraging to see this important issue being addressed. Hemophilia doesn’t directly cause iron deficiency, but heavy menstrual bleeding can lead to iron loss. Women with hemophilia who experience heavy menstrual…
Review study shows prevalence, impact of hemophilia in Brazil
In Brazil, hemophilia markedly impairs patients’ quality of life and ability to work, with up to 82.6% of people with the disorder being unemployed, a review study finds. Barriers to treatment include long distances to centers, lack of coordination of specialized and emergency care, and restricted access to rehabilitation.
Kogenate FS (octocog alfa) for hemophilia
Last updated May 29, 2024, by Lindsey Shapiro, PhD Â Fact-checked by Joana Carvalho, PhD What is Kogenate FS for hemophilia? Kogenate (octocog alfa) was an approved treatment used to prevent bleeding episodes, including those occurring during surgery, in people with hemophilia A that has now…
FDA OKs clinical testing of BE-101 as potential hemophilia B treatment
The U.S. Food and Drug Administration (FDA) has cleared clinical testing in adults of BE-101, a B-cell treatment candidate for hemophilia B being developed by Be Biopharma. The Phase 1/2 trial, called BeCoMe-9, will be a first-in-human study designed to assess the safety and preliminary efficacy of BE-101…
How do you thrive with hemophilia in a small town? Get involved
I live in the small town of Punta Gorda, Florida. Though I have hemophilia B and von Willebrand disease, I don’t know anyone here or in the surrounding Charlotte County with a bleeding disorder. While living in such a community can present challenges when dealing with…