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.
Learn more
Resources
Steve on gene therapy for haemophilia
Steve shares his experience having gene therapy for severe haemophilia A – and how it has changed his life
Gene therapy booklet
Gene therapy for haemophilia Information for people with haemophilia about gene therapy and how it works in haemophilia. This booklet explains the types of gene therapy that are being trialled in haemophilia at present, who...
Gene therapy for haemophilia fact sheet
This fact sheet answers how gene therapy for haemophilia works, who can have it, the risks and benefits, the treatment process and other FAQs.
Haemophilia gene therapy snapshot survey
HFA’s haemophilia gene therapy snapshot survey asked people and parents of children with haemophilia what outcomes they would like from treatment now and in the future.
Haemophilia Treatment Preferences Survey
(HaemPref Survey) How do individuals make decisions about treatment? What factors encourage or discourage patients from treatments? What are individuals’ treatment goals? Results presented are based on a study conducted by Community and Patient Preference...
Gene and emerging therapies in haemophilia
Exciting advancements in gene therapy and other emerging treatments for haemophilia were presented at the Blood 2024 conference.
Prof Cedric Hermans on New Haemophilia Treatments and more
Prof Cedric Hermans spoke with HFA about new treatments for bleeding disorders and the importance of diagnosis for women and girls, and the broader community
Gene Therapy Interview with Dr Michiel Coppens
In this interview chaired by Dr Stephanie P’ng and led by questions from the community, Dr Michiel Coppens explains what gene therapy is, how it works, and how it can be used to treat people...
Gene and emerging therapies fact sheets
Clotting factor therapy, bispecific antibody therapy, rebalancing therapies and gene therapy factsheets are now available
Dom on having Gene Therapy for Haemophilia A
Dom shares his experience of receiving gene therapy treatment for haemophilia A
More information
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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