It is an exciting time in gene therapy for haemophilia in Australia.
Gene therapy for haemophilia aims to provide the body with a functioning version of the factor VIII (8) or factor IX (9) gene so that the body can produce enough clotting factor for the blood to clot normally.
Gene therapy has been registered for use in some countries. In Australia there are currently a number of advanced experimental clinical trials for gene therapy. Some Haemophilia Treatment Centres are participating in the trials. People who are interested can discuss this with their Haemophilia Treatment Centre as this is constantly changing.
Gene therapy for haemophilia
This resource, Gene Therapy for Hemophilia is published by World Federation of Hemophilia (WFH) and has been linked here with permission. © 2023 World Federation of Hemophilia https://sdm.wfh.org/
Gene therapy is still a relatively new treatment for haemophilia and there is a lot of research in this area and new information becoming available. Initial results are promising, with a decrease in the number of bleeding episodes and the need for people to have prophylaxis treatment.
There are a lot of important considerations before undertaking gene therapy, for example, will you be suitable to have gene therapy, how long will the effect of treatment last, side effects, etc. All of these need to be discussed with your Haemophilia Treatment Centre.
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For more information on gene therapy in haemophilia, speak to your treating haemophilia specialist doctor or a gene therapy expert who is undertaking clinical trials in haemophilia.
Click here for contact details of your local specialist Haemophilia Treatment Centre.
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