World Impact 

Sasha’s treatment isn’t just a breakthrough for one child, 

it’s a turning point for medicine.

First of Its Kind

Genetic Therapy

Top scientists in Australia and the US are developing a personalised Antisense Oligonucleotide (ASO) to treat Sasha’s unique splice site mutation in the SLC6A1 gene. SLC6A1 is a type of genetic epilepsy. This is the first of it’s kind cure.
This breakthrough is Sasha’s only chance to speak, learn and live independently.

Sasha’s Cure Will Help Millions

Sasha the Pioneer

Your donation is part of a much bigger picture. Sasha has a splice site mutation (ie, a mutation that affects RNA splicing). 15%+ of all disease-causing mutations are splicing mutations! So when we show that Sasha's mutation can be fixed, that will pave the way to treating millions of people for all kinds of genetic diseases.

This is our only chance to help our daughter. We must fight for her, and millions of other children like her.

A young girl with curly hair sitting in a stroller, wearing a gray t-shirt, outdoors on a sunny day.

True Hope for Other Families

For families facing rare, untreatable genetic diseases, Sasha’s journey represents something extraordinary: proof that it’s possible. Until now, children with splice site mutations were told there was no treatment — that their disease was too rare to matter. Sasha’s ASO challenges that outdated narrative. Her treatment will show the world that no child is too rare to save.

Hear from Professor Sue Fletcher, AO about Saving Sasha

  • Once we prove this is safe and effective for Sasha, we set a precedent. Sasha’s treatment opens doors, not just for SLC6A1 patients, but for countless others with RNA splicing mutations in genes across the human genome.

  • Sasha’s journey is driving global collaboration between leading scientists, regulatory bodies and rare disease advocates. It’s accelerating the path to smarter drug development — faster, safer and more targeted.

  • Sasha’s mutation is unique to her — but the implications of her treatment are global. Her personalised ASO is the first ever created to treat a splice site mutation. Her cure will show that medical science can precisely target the underlying cause of countless rare diseases, not just treat symptoms.

By developing the world’s first antisense oligonucleotide (ASO) for a splice site mutation, scientists are proving what’s possible for rare genetic conditions once thought untreatable.

Three people sitting outdoors on grass, smiling, with a woman on the left, a man on the right, and a girl in the middle holding a phone, with a quote about Sasha and genetic medicine.

Sasha’s Cure

is Shaping the

Future of

Genetic Medicine

A circular badge with the text 'Global First' at the top and 'World's First Aso for Splice Site Mutation' at the bottom. It features a side profile of a young girl with short hair and a double helix DNA strand in front of her face.
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