WELCOME TO THE

Gene & Emerging Therapies Hub

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.

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Fact sheet / Booklet

Gene therapy

AAV vectorsclinical trialsgene therapyhaemophilia

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...

 News & Stories

HFA Gene Therapy Snapshot Survey

HFA would like to understand what our community members want from their haemophilia treatments and your perspectives on gene therapy in particular.

 National Haemophilia Journal

Gene therapy

gene therapyhaemophilia

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...

 National Haemophilia Journal

Haemophilia gene therapy snapshot survey

gene therapyhaemophiliaoutcomestreatment

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.

 National Haemophilia Journal

MyABDR and recording your treatment

MyABDRtreatment recording

With new haemophilia treatments, recording on MyABDR has some challenges. HTCs and users share their tips.

 National Haemophilia Journal

Novel haemophilia therapies

acquired haemophiliafitusirangene therapyhaemophiliaHemlibranon-factor therapytreatment

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...

 National Haemophilia Journal

World Hepatitis Day 2024

bleeding disordersgene therapyliver healthWorld Hepatitis Day

World Hepatitis Day 2024 reminds us It’s time for action – hepatitis C and liver health remain important issues for our community and acting now is vital.

 National Haemophilia Journal

Jack’s latest travel adventures

adventure sporthaemophiliatravelYouth

Jack talks about his travel adventures in Canada and the USA, snowboarding and managing haemophilia on the road with new treatments.

 National Haemophilia Journal

New haemophilia therapies

EHLsgene therapyhaemophiliaHemlibramonoclonal antibodiesrebalancing therapiestreatment

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....

 National Haemophilia Journal

From the President

Damon Courtenay Memorial Endowment Fund (DCMEF)gene therapyHepatitis CrepresentationSouth Australia

As we wrap up 2024, HFA reflects on our achievements in new therapies, hep C advocacy and support during the year.

 National Haemophilia Journal

ISTH 2024 Congress: novel therapies

emerging therapieshaemophiliatreatment

Exciting new haemophilia treatments using a range of innovative technologies were highlighted at ISTH 2024 Congress.

 National Haemophilia Journal

Haemophilia treatment survey

EHLsgene therapyhaemophiliaoutcome measuressurveystreatment

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...

Information

Gene therapy FAQs

Is gene therapy a cure for haemophilia? In many cases successful gene therapy means that a person with severe haemophilia now has mild haemophilia, although a small number now have normal factor levels. It is...

 News & Stories

Have your gene therapy questions answered

webinar

Dr Michiel Coppens is coming to Australia and we want your questions for a special ‘Ask Me Anything’ interview about gene therapy.

 Video

New Therapies Webinar

gene therapy

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...

 National Haemophilia Journal

New haemophilia therapies

gene therapyhaemophilianon-factor therapy

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 ~...

 Video

Gene Therapy Interview with Dr Michiel Coppens

gene therapy

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...

 National Haemophilia Journal

Gene therapy webinar

gene therapyhaemophilia

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.

 News & Stories

Haemophilia – the spice of life

careermusictransition

Willem spoke to us about working, exercising and being out and about in these days with new treatments.

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.

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