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Research at The Florey

​The path to developing a new medicine is a long and expensive journey which the ANPDF is committed to traveling with The Florey.

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Research Goals + Progress

From Lab to Lifesaving: Our hope for the future of Niemann-Pick disease type C

It is estimated from Discovery to Clinical Trials to take 10+ years and USD 94 million.

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With your support, the goal is to move into the second stage of this process within the next 12 months, bringing us one vital step closer to offering a local and life-changing treatment to Aussies suffering from Niemann-Pick disease type C.​


Developing an mRNA gene therapy for Niemann-Pick Disease Type C

Current research projects in the Oxidation Biology Unit at The Florey Institute of Neuroscience and Mental Health (Melbourne, Australia) are led by Dr. Ya Hui Hung and Professor Ashley Bush.


The Australian NPC Disease Foundation strongly supports this research to find new treatment options that will provide better health outcomes for NP-C patients both in Australia and around the world. ​​​


Their research currently focuses on screening drug candidates using NP-C patient cells. There is excellent progress in identifying drug candidates that can restore cholesterol processing, which may lead to new treatment options for NP-C.

Useful Resources

For Families

  • New funding boosts treatment for ultra-rare childhood dementia - Link

    • Developing an mRNA gene therapy for Niemann-Pick Disease Type C - Link

For Clinicians and Researchers

  • Meet the lead Researcher, Dr Ya Hui Hung at The Florey - Link

  • How to Apply at The Florey - Link

  • Developing an mRNA gene therapy for Niemann-Pick Disease Type C - Link

General

  • Minister Butler's Announcement, Nearly $400 million for exceptional medical research projects including projects to improve First Nations health - Link


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