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At just two weeks old, Alessia was diagnosed with Spinal Muscular Atrophy (SMA)—a condition that would have taken her ability to move, breathe, and ultimately, her life. Thanks to ground-breaking gene therapy, Alessia became the first child in Australia to receive life-saving treatment, giving her a future her parents thought they might never see. 

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Today, Alessia is thriving, full of curiosity and a love for puzzles, a living testament to the power of research. 

But not every child with SMA has access to this treatment. Some children cannot receive gene therapy due to natural immunity, while others with other deadly conditions face the heartbreaking reality that there is no treatment.

Our researchers at Children’s Medical Research Institute are developing the next generation of gene therapy—one that can bypass natural immunity—ensuring that no child is left behind. 

But we cannot do it alone.

Your gift today will fund vital research that transforms lives. Together, we can make sure more children, like Alessia, have the chance to grow, play, and celebrate milestones with their families.

Help Fund the Future

Your donation is more than a gift—it’s hope. Hope for families who want a future for their children. Will you give that hope today?

Together we can beat children's genetic diseases.

Select your donation amount:

Fund gene sequencing to identify changes in DNA that cause disease
Help us bring SMA cures to more kids like Alessia
Support advances in gene therapy so more kids have access to cures

Accelerate the search for cures.

By donating monthly, you can fund research that will have an even bigger impact on children living with a genetic disease.