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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 a global leader in providing crucial support for research and education necessary to accelerate cures 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?

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


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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.

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Latest News & Events
Firefly Fund Announces $5 Million Gift

FOR IMMEDIATE RELEASE February 27, 2024 Firefly Fund Announces $5 Million Gift to Create Center for Rare Disease at UT Austin Dell Medical School Austin, Texas – To mark World…Read More

Texas Infant to Become Fifth Child to Receive Life-Saving Gene Therapy Treatment on U.S. Soil

DALLAS, Feb. 05, 2024 (GLOBE NEWSWIRE) — Sufyan Pashai, a 4-month-old from Dallas, will travel to Minnesota and become the fifth child to receive life-saving gene therapy treatment on U.S….Read More




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