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.
New Therapies Webinar
Dr Liane Khoo (Haematologist and HTC Director, Royal Prince Alfred Hospital), Dr Sally Campbell (Haematologist, and acting HTC Director, Royal Brisbane & Women’s Hospital), Stephen Matthews (Clinical Nurse Consultant, HTC, Royal Prince Alfred Hospital) and Sharon...
New haemophilia therapies
ALEX KLEVER Alex Klever is Clinical Nurse Consultant – Haemophilia at the Queensland Haemophilia Centre, Royal Brisbane & Women’s Hospital Plenary – Treatment landscape into the future Chair ~ David FaganFrom then to now ~...
Gene therapy interview with Dr Michiel Coppens now available
On 14 May 2024 we were fortunate to have Dr Michiel Coppens join us at the HFA office for an interview about gene therapy. Watch it now.
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 therapy webinar
Dr Michiel Coppens joined us at HFA for an interview about gene therapy, answering questions from the community. Watch the video or read the transcript.
Congress – a Foundation perspective
Haemophilia Foundation Australia President Gavin Finkelstein spoke with HFA about his experiences at the WFH 2024 World Congress.
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.
Sources and reviewers