1 IN 20 KIDS


Right now, we’re working on gene therapy cures for previously incurable genetic diseases, but we need your help.

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The 1 in 20 kids facing a birth defect or genetic disease are used to hearing words like “incurable” or “lifelong effects”. It doesn’t have to be this way.

Gene therapy is a method of curing genetic diseases by correcting the cause, by fixing the error in a child’s DNA.

Our researchers at Children's Medical Research Institute have helped bring gene therapy cures to Australia for “boy in the bubble disease” (SCIDX1-deficiency), spinal muscular atrophy, and the cure we’ve developed for a metabolic liver disease (OTC-deficiency) is entering clinical trials in the UK.
With your help we can do so much more.
We are establishing a new gene therapy initiative to fill the gap in Australian medicine. We want to ensure children facing genetic diseases go from diagnosis to cure, and that those cures are available right here at home.

No child’s life should be cut short, and we will do all within our power to change “incurable” to “curable”.

You're helping kids like Ryan

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