FROM ONE TO MANY Advancing Precision Therapies for Children with Rare Diseases

Transforming one child’s treatment into solutions for many

Join us for a powerful evening dedicated to transforming the future of rare disease research for children in Queensland.

This special event will bring together families, scientists, clinicians, and supporters to accelerate the development of life-changing personalised therapies for children who currently have no treatment options.

On the evening, Dr Laura Croft will share how cutting-edge genetic therapies-already transforming lives overseas-can be developed locally for Australian children. You will also hear from Sasha’s family, who are working with us to develop a mutation specific personalised ASO treatment for Sasha’s ultra-rare genetic epilepsy, caused by a single mutation in her SLC6A1 gene, and from Eliza, mum to a daughter with a treatment resistant epilepsy.

95% of rare diseases have no approved treatment. Every month without treatment can mean continued disease progression. Your support will help build a pathway to deliver personalised therapies for children who would otherwise be left behind.

Join us. Support change. Transform lives.

Three-course dinner | Inspiring speakers | Raffle tickets

Contact the host of From One to Many - Advancing Precision Therapies for Children with Rare Diseases

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