Shining a Light on NPC
Our mission is to fund and support the research and education necessary to accelerate a cure for rare neurodegenerative genetic diseases that affect children and currently have no cure. We have begun our journey with a focus on Niemann-Pick Disease Type C.
Donate Now
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 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
Share Your Story
We'd Love to Hear From YouLatest News & Events
Firefly Fund Commits $500K to Novel Newborn Screening Study That Will Help Identify Rare Diseases at Birth
FOR IMMEDIATE RELEASE: May 10, 2021 CONTACT: Pam Andrews pam@fireflyfund.org Austin Nonprofit Commits $500K to Novel Newborn Screening Study That Will Help Identify Rare Diseases at Birth  Pilot Study enrolls…Read More
Firefly Chat Episode 7 (Part 2): NPC Moms React to Adrabetadex News
A continuation of Firefly Fund co-founder Pam Andrews’ discussion with NPC moms, highlighting the benefits of Adrabetadex. The moms share their fears about a future without the medication, the popular…Read More