The treatment landscape for bleeding disorders is changing
It is an exciting time in gene and emerging therapies for bleeding disorders in Australia. The aim of the Gene & Emerging Therapies (GETs) hub is to give the bleeding disorders community up-to-date information and knowledge about all these different treatments and to help make a shared decision with family and your HTC as they become available.
Gene therapy
Other new and emerging treatments
Making decisions about treatment
Information for health professionals (coming soon)
Resources
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.
Bispecific antibody therapy fact sheet
This factsheet on bispecific antibody therapies explains factor VIIIa (8a) mimetics like emicizumab, how they work and are given in haemophilia.
Steve on gene therapy for haemophilia
Steve shares his experience having gene therapy for severe haemophilia A – and how it has changed his life
Rebalancing therapies fact sheet
This factsheet explains how rebalancing therapies work in haemophilia and are given, types (TFPIs, siRNAs) and side effects.
WFH Summit: new therapies and approaches
WFH CCS topics included health literacy in shared decision-making, new therapies for VWD and women and girls with bleeding disorders.
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
ISTH 2024 Congress: novel therapies
Exciting new haemophilia treatments using a range of innovative technologies were highlighted at ISTH 2024 Congress.
Keeping your liver healthy
A healthy liver is important to us all. Your liver has a lot of different jobs to do to keep your body working well. These are some tips from our liver specialists to keep your...
From the President
So much is happening and changing very quickly – hepatitis C, women and girls, the conference and new therapies.
Prof Cedric Hermans on new haemophilia treatments and more
Professor Cedric Hermans spoke with HFA about new and emerging haemophilia therapies, women and girls, and the importance of diagnosis.
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...
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.
World Hepatitis Day 2026
World Hepatitis Day is a reminder that hepatitis C and liver health remain important issues for our community.
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...
Haemophilia – the spice of life
Willem spoke to us about working, exercising and being out and about in these days with new treatments.
ISTH 2025 – emerging therapies in haemophilia and VWD
At ISTH 2025 researchers shared advances in emerging therapies for haemophilia and von Willebrand disease (VWD).
Acknowledgments
The Gene and Emerging Therapies Hub has been funded by education grants from CSL Behring and Sanofi. This funding is based on a mutual understanding that HFA will act independently for the benefit of the bleeding disorders community. HFA does not promote any particular pharmaceutical product. Where possible products are referred to by their class or generic name and any mention of any commercial brand is strictly for educational purposes.