LIVE LIFE FOUNDATION
What We Do
At Live Life Foundation, our mission is to raise $4.5M for Australia’s first gene therapy ward at the Royal Children’s Hospital.
UNDERSTANDING THE SCIENCE
What is gene therapy?
Gene therapy, a type of advanced therapy, replaces defective or missing genes in patients’ cells with a healthy version. For this treatment to work, a large dose of the healthy gene must reach enough affected cells. This is a huge challenge, as the human body has around thirty trillion cells.
Conditions gene therapy can help
Spinal Muscular Atrophy (SMA)
A rare genetic condition causing muscle weakness and movement loss, often affecting breathing and mobility.
Duchenne Muscular Dystrophy
A progressive muscle-wasting disorder that leads to severe physical disability, mostly in young boys.
Leukaemia
A type of blood cancer that affects the bone marrow, impacting the body’s ability to fight infection.
Stroke
A sudden interruption of blood flow to the brain that can cause lasting physical and cognitive impairment.
Haemophilia
A rare bleeding disorder where the blood doesn’t clot properly, leading to excessive internal bleeding.
Parkinson’s Disease
A progressive nervous system disorder that affects movement, balance, and muscle control over time.
Epilepsy
A neurological condition that causes recurring seizures, which can impact learning, safety and development.
Live Life Foundation
Why Does Gene Therapy Matter?
Genetic conditions affect around one in 400 children, with one in five paediatric hospital admissions linked to a genetic disorder. These conditions have a profound impact on the child, their family and on the community.
“SMA, also known as spinal muscular atrophy, a devastating condition where untreated children often do not survive past two years of age. However, if this condition is treated before symptoms begin to arise, these children will have their best chance of enjoying a completely healthy, normal life”.