Australian girl becomes first to survive childhood dementia through gene therapy
Tilly Page, nine, is the only Australian child to receive gene therapy for childhood dementia and shows no signs of disease.
Tilly Page was born with Sanfilippo syndrome, a genetic form of childhood dementia that kills nerve cells in the brain. Half of children with this condition die before their tenth birthday.
At three months old, her parents were told to take her home and make memories. At one year old, she received experimental gene therapy, the first Australian child to do so and the youngest in the world.
Today, nine years later, doctors say there is no evidence the disease has progressed. Tilly plays netball, reads to her class and writes thank-you letters.
Geneticist Dr Nick Smith developed the therapy with pharmaceutical company Ultragenyx. The treatment uses a virus to deliver a working copy of the faulty gene into brain cells.
- before age ten
- Half of children with Sanfilippo die
- one year old
- Age at treatment
- nine years old
- Current age
- Dr Nick Smith, Adelaide
- Geneticist
Why it mattersA child given weeks to live is now thriving, offering hope to other families facing childhood genetic disease and showing gene therapy can prevent fatal progression.
AustraliaOne Australian breakthrough offers a pathway for other children with rare genetic conditions and demonstrates Australia's capacity to run cutting-edge medical trials.
✓ Claims checked against the source and corrected before publish. checked 11 d ago
