Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment

https://blog.cirm.ca.gov/2025/03/11/breaking-barriers-in-rare-disease-a-gene-therapy-transforms-cystinosis-treatment/?fbclid=IwZXh0bgNhZW0CMTEAAR1f2iMPF-5wFuiZ8wCpPuhUuf7zLV6YMj8IsPSFiAXufzKO-IBdsuNokhw_aem_GQWXMpTV8eYttvKdR13VfQ

Acknowledgement of Country

The Australian Cystinosis Foundation acknowledges the Traditional Custodians of country throughout Australia and their connections to land, sea and community. We pay our respects to them and their cultures and elders past, present and emerging.

The Australian Cystinosis Foundation is an independent not-for-profit registered charity dedicated to helping Australian patients with Cystinosis and their families. Through support, education, patient services and collaboration with medical professionals.