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

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.

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.

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.

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.

The Children’s Hospital at Westmead has launched a world-first clinical trial investigating the use of phage therapy to treat a chronic bacterial infection in children with cystic fibrosis (CF).

The Rett syndrome clinic at Westmead, a world leader in its field, is looking towards gene therapy research to treat the condition – giving hope to families like Abigail's for a brighter future.

Three boys in NSW have become the youngest in the world to receive therapy for Duchenne muscular dystrophy in a world-first trial.