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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Resources
Gene therapy
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...
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.
From the President
Heading to the end of the year, we review our progress. Gene and emerging therapies are on the agenda, and Bleeding Disorders Awareness Month is coming up.
Gene therapy
Gene therapy for haemophilia has been in advanced clinical trials for some years now and this Congress was a timely forum to discuss and evaluate it critically. ROBYN SHOEMARK Robyn Shoemark is Clinical Nurse Consultant...
Novel haemophilia therapies
Living in the new world of novel haemophilia therapies ROBYN SHOEMARK Robyn Shoemark is Clinical Nurse Consultant Haemophilia/Haematology at the Kids Factor Zone, The Children’s Hospital at Westmead, Sydney, NSW Discussion about the impact of...
From the President
As we wrap up 2024, HFA reflects on our achievements in new therapies, hep C advocacy and support during the year.
New haemophilia therapies
It is an exciting time with more new haemophilia treatments likely to become available in Australia, but there have been a lot of questions about them and what they will mean for treatment and care....
Haemophilia treatment survey
SUZANNE O'CALLAGHAN Suzanne O'Callaghan is HFA Policy Research and Education Manager What information does our community want about new haemophilia treatments? And for those who are affected, what would you like haemophilia treatment to achieve...
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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