Firefly Fund: Shining a Light on NPC

Firefly Fund is a global leader in providing support, resources, and reassurance to families living with rare neurodegenerative genetic diseases like Niemann-Pick Disease Type C that affect children and currently…Read More

Dan Rather speaks on behalf of the Firefly Fund

Please consider making a donation at Firefly.Fund/JoinTheFlight. Your donations fund new drug therapies that can slow the progression of Niemann-Pick Disease Type C and other neurodegenerative diseases that cut short…Read More

What is NPC?

Ahead of World Rare Disease Day 2020, Firefly Fund’s founders and researchers explain about Niemann-Pick Disease Type C (NPC) and how you can join the flight to help fund and…Read More


View All Videos
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.


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

Share Your Story
We'd Love to Hear From You
Latest News & Events
Firefly Chat Episode 4: Newborn Screening

This month’s episode of Firefly Chat focuses on Newborn Screening Awareness Month. The podcast was repurposed from a discussion at the recent World Orphan Drug Congress, featuring speakers Firefly Fund…Read More

Firefly Chat Episode 3 (Part 2): Orphazyme

Tune in for the second installation of Firefly Fund Co-Founders Pam & Chris Andrews’ conversation with Thomas Kirkegaard Jensen Ph.D., Co-Founder and Chief Scientific Officer and Kim Stratton, CEO of Orphazyme, a…Read More




View All News & Events
$519,000
Raised
Our Fundraising Goal

As of July 1, 2020 Firefly Fund has raised $519,039.65 towards its 1M goal for 2020.

Make a Donation

How You Can Help
Get Involved