About This Disease

Niemann-Pick Disease Type C is a progressive and neurodegenerative genetic fatal disease caused by a buildup of cholesterol in the brain, liver and spleen.


About This Disease

Until more research and treatment are available, the majority of children afflicted with NPC will die before age 20.


About This Disease

Symptoms of NPC are hard to detect. Diagnosis often takes years.


About This Disease

Symptoms include ataxia, the balance disorder that causes clumsiness and frequent falling; developmental delay; difficulty swallowing; vertical gaze palsy, or difficulty moving the eyes rapidly up and down; and enlargement of the liver or spleen.


About This Disease

Currently, researchers are engaged in a clinical trial testing the effectiveness of a promising new drug, VTS-270.


Firefly Fund: Shining a Light on Rare Disease

Firefly Fund is committed to advancing and accelerating access to life-saving and disease modifying therapies for rare genetic diseases that affect children and currently have no cure.

Dan Rather speaks on behalf of the Firefly Fund

Your donations fund crucial support for research and education necessary to accelerate cures for rare genetic diseases that affect children and currently have no cure.

What is NPC?

Firefly Fund’s founders and the researchers from the scientific community share an overview of Niemann-Pick Disease Type C (NPC) inviting supporters to help advance the organization’s mission to accelerate a…Read More


Belle and Abby's Story

On March 10, 2016, Pam and Chris Andrews were told their daughter Belle, only six years old, would be unlikely to live past her teenage years. It’s a parent’s worst nightmare. No, it’s worse than that. Because NPC is such a rare disease that no parent would even know to fear it could happen to their child. NPC is also a genetic disease, so shortly after Belle’s diagnosis, the Andrews’ nightmare was magnified when they found out their other daughter, Abby, had inherited the disease, too. Abby is only two years old.

Read the Full Story

Latest News & Events
OpEd: Proposition 14 Offers Hope Against Dementia for Families

October 10, 2025 | Austin American-Stateman Check out today’s Austin American-Statesman OpEd by Firefly Fund co-founders Pam and Chris Andrews. Proposition 14 is a vote for compassion, innovation and Texas…Read More

Pam & Chris Andrews featured in RARE Revolution

September 8, 2025 | We’re excited to share a new interview with Pam & Chris Andrews in RARE Revolution Magazine. A huge thank you to Nicola Miller for capturing our…Read More


How You Can Help