News

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.