07/09/2026
This World Duchenne Awareness Day, your support is helping bring emerging treatments closer to children living with rare diseases.
At The Children’s Hospital at Westmead, a world-first international clinical trial is exploring whether a single dose of gene therapy could target Duchenne at its root cause by replacing the faulty gene responsible for the condition.
DMD is a rare and life-limiting genetic condition that almost exclusively affects boys, causing progressive muscle weakness, with almost all needing a wheelchair by the age of 12.
Supported through the Kids Advanced Therapeutics (KAT) program, the trial is helping bring cutting-edge treatments to children while building the expertise needed to deliver advanced therapies in paediatric care.
Your support of Sydney Children's Hospitals Foundation is helping experts continue exploring what’s possible for children with rare and complex conditions.