Accelerating discoveries for rare diseases through gene therapy

30 Nov 1999

Your support is helping researchers from the Network, along with partner organisation, the Children's Medical Research Institute, to accelerate world-leading gene and gene-modified cell therapies to help children with rare diseases. 

Gene therapy involves a once-only injection and has the potential to transform the lives of children.

Expert clinicians and families have witnessed the incredible benefit of giving Zolgensma to children with spinal muscular atrophy, a devastating life-threatening condition that leads to the rapid loss of motor function in the first year of life. Children receiving this therapy are now walking and developing normally at age two.

Another example is ocular gene therapy, which involves an injection into the retina of children who are deficient in the RPE65 protein and at risk of blinding eye disease. The therapy has lead to the partial restoration of the vision in two teenagers allowing them to see stars for the first time in their lives.

Leading researchers have unique opportunities to develop novel gene therapies focused on rare genetic diseases of the liver, eye and brain – the latter of which can lead to childhood dementia.

Related Posts

The youngest and brightest minds are searching for answers

Research Spotlight

Partnering for change in Aboriginal child health

NETS Stats

Professor Chris Forrest bringing together SCHN and Paediatric Plastic Surgeons

SCHF brought World renowned plastic surgeon Professor Chris Forrest from Toronto and he is...

Kids Advanced Therapeutics

A Movement of Many, fighting for the health and wellbeing of all children in an ever changing world.

See how the Movement of Many are helping sick kids

See your impact