A world-first clinical trial using a novel liquid biopsy has transformed the lives of children and young people with arteriovenous malformations (AVMs).

Kids Research showcased the power of collaboration at the Kids Advanced Therapeutics Symposium bringing together healthcare providers, researchers, industry leaders, community partners and families.

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.

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.

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

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).