This project conducted at The Florey will develop an effective mRNA gene therapy that will significantly improve the health and well-being of those living with NP-C. Findings from this project will enable us to rapidly build an mRNA gene therapy pipeline to treat other genetic disorders, in particular those presenting with childhood dementia.

This nationwide collaborative project aims to improve the standard of care for children and families impacted by Niemann-Pick disease type C (NP-C). Our project will create resources to equip clinicians and the community to better identify, diagnose and effectively manage this severe, life-limiting disease. Through the adaptation of 2018 international guidelines with Niemann-Pick specialists and the community, we will create knowledge-building resources that make a positive impact on the lives of people living with NP-C.