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.

Patients with difficult-to-treat bacterial infections across NSW will soon have better access to limb and life-saving therapy known as phage therapy, thanks to a $3.5 million funding boost announced by the NSW Government.

A world-first clinical trial has been launched at Sydney Children’s Hospital, Randwick for children with ependymoma – a rare and devastating form of paediatric brain cancer.

Steps are being taken by SCHN to improve equity of access and service management for those living with a rare disease via The Navigator Project.

The development of a world-first nasal spray vaccine could be a turning point in protection against whooping cough, helping stop the spread of the respiratory infection to vulnerable babies.