In Australia today, there are over 400,000 children who have a rare disease. It’s a surprising statistic, but while rare diseases are rare individually, there are in-fact over 7000 rare diseases, with many more discovered every year. 

There is no current cure available for 95% children born with a rare disease, and 30% of these children will tragically die before their fifth birthday.  

Advanced Therapeutics, like cell and gene therapy, biologics, and other personalised medicines/devices, hold the potential to transform the lives of children with rare genetic and acquired disorders. 

SCHN is recognised as a leading paediatric centre for Advanced Therapeutics in Australia. Known as the KAT (Kids Advanced Therapeutics) Program, clinicians and researchers are working to translate Advanced Therapeutics research into clinical practice across SCHN, with key roles designed to bridge research and clinical operations, build research capability and ensure integration and application of Advanced Therapeutics into future service models. 

Advanced Therapeutics at SCHN has already seen a world first successful trial for kids under four with Duchenne Muscular Dystrophy, which used gene replacement therapy to target the disease at its roots.  

Future trials can provide a child with earlier access to treatment for a range of conditions including cancer, epilepsy, neuromuscular and neurodevelopment disorders, metabolic disorders, genetic deafness, and blindness. The KAT Program offers life-changing therapies for children whose conditions so far have not been able to be treated.  

With philanthropic support, The KAT Program will enable children of NSW and Australia to access these potentially transformative therapies through the delivery of specialised paediatric clinical trials at SCHN. 

The KAT program has the potential to transform the lives of children with rare diseases but there is an ongoing and urgent need to increase the resources available to support it. With your help we can empower these dedicated researchers and clinicians to do more research, help more children and create a better future. 

Contact andre.brady@schf.org.au to learn more about how you can be involved in translational research and transformative care.