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About This Disease

Niemann-Pick Type C1 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 NPC1 will die before age 20.

About This Disease

Symptoms of NPC1 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 NPC1 is such a rare disease that no parent would even know to fear it could happen to their child. NPC1 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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Step up to the Tee Fore Their Future!

EXCLUSIVE, SIGNED GOLF BAG  UP FOR AUCTION Want to make a difference TODAY? You can light the way of the Firefly Fund by participating in an online auction for a…Read More

Andrews family featured in Austin American-Statesman

“Dell Children’s Medical Center of Central Texas has been participating in an international study testing a treatment for Niemann-Pick Type C1 disease called VTS-270. We wrote about the trial in November, when…Read More

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