A new trial medication has effectively saved Xavier's life as someone with spinal muscular atrophy.

Researchers at Sydney Children’s Hospital, Randwick have found a new testing method supporting families living with Tuberous Sclerosis Complex.

At eleven years old, John is a bright and bubbly pre-teen who lights up every room with his infectious smile. But behind it all is a diagnosis that changed his life and a journey marked by his courage to pursue something more.

Ten-year-old Mateo was able to receive life-saving treatment through a clinical trial after suffering a sudden cardiac arrest.

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.

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

In an Australian-first clinical trial, a novel full-spectrum cannabinoid drug therapy has shown to have life-changing impacts for those with Rett syndrome.

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 carb, 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).

As Professor Joshua Burns PhD bids farewell to his time at the Sydney Children’s Hospitals Network, he leaves behind a 20 year long legacy of incredible work and transformative leadership.