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Thanks to the work by the Kids Advanced Therapeutics program, we were the first site to dose participants in a worldwide SMA gene therapy clinical trial.

For families like Angelina’s, a rare disease diagnosis can feel like being lost in a dark forest, desperately searching for a clear path forward.

Precision medicine offered a renewed hope for ten-year-old Nicholas when his acute myeloid leukaemia returned.

It would take 10 years before genomic testing would finally diagnose Edmond with an extremely rare neuropathy and support in providing a treatment with life-changing results.

Four talented researchers have become the first to receive our new clinician researcher fellowships at Kids Research.

Researchers and honoured guests of the Kids Neuroscience Centre (KNC) were joined by patients and families at Government House to celebrate 25 years of innovative research.

Our new CRC offers comprehensive care during clinical trials in one location, providing a more comfortable and welcoming environment for patients and staff.

A world-first study has shown a low carbohydrate, high fat diet could have a life-changing impact on cognitive function for children living with Kabuki syndrome.

SCHN investigators have been awarded a grant to research the specific types of immune cells involved in FPIES reaction to potentially improve diagnosis and treatment.

An NHMRC Partnership Grant worth $1.5 million has been secured to investigate the impacts of current genetic therapies for Inherited Retinal Diseases (IRDs).