The report, A Rare Kind of Care, examines the current landscape of rare disease policy in Australia, aimed at ensuring all living with a rare disease experience health equity.

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.

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.

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.