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.

Doctors in the Kids Cancer Centre at Sydney Children’s Hospital, Randwick will use CAR T-cell therapy to try to attack and destroy childhood brain cancer.

Babies diagnosed with Spinal Muscular Atrophy at birth who received life-changing gene therapy are now living longer and healthier, celebrating their first birthday symptom-free or with minimal symptoms thanks to the ground-breaking research by Kids Research.

New trial investigates the occurrence of food allergies in children amid dramatic rise in the number of children diagnosed with food allergies.

At four months of age Sophia is reaching all her developmental milestones. She demonstrates good head control while sitting supported and can even roll from her tummy to her back.