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.
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
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Firefly Fund’s Pam and Chris Andrews Featured on Austin Next Podcast
October 2, 2024 (AUSTIN NEXT) Pam and Chris Andrews joined Jason Scharf’s podcast, Austin Next, a podcast exploring emerging trends, technologies, and their convergence. The episode highlights Firefly Fund’s vision…Read More
Austin Family Optimistic After FDA Approves New Drugs
October 6, 2024 (FOX) – Two Austin sisters are fighting a rare genetic disorder, but the recent FDA approval of two new drugs is offering the family a promising path forward….Read More
