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.

As a part of the Navigator Project, a targeted nurse-led support service has helped Finn's family in navigating a rare disease diagnosis and treatment.

A new viral vector manufacturing facility being built in the Westmead precinct is set to change the treatment landscape for patients with genetic diseases, cancers and viral infections, enabling gene therapies to be trialled and delivered sooner.

The latest round of MRFF grants will further drive innovation and research across Kids Research into areas such as childhood asthma, cancer, Cerebral Palsy and trauma care.

The groundbreaking FIREFLY-1 trial tested the efficacy of oral drug, tovorafenib, in brain tumour patients with results showing a 50 per cent tumour shrinkage in two thirds of patients.