Firefly Fund is a 501(c)3 nonprofit organization founded in 2017 to fund the research and education necessary to accelerate a cure for rare neurodegenerative genetic diseases that affect children and currently have no cure — starting with Niemann-Pick Disease Type C (NPC). Headquartered in Austin, Texas, Firefly Fund leads a collection of programs including Newborn Screening, Translational Medical Research, and the Patient Access Fund. These programs offer resources, support, and reassurance to NPC families. A rare disease diagnosis is unimaginable, but together we will find a cure.
Firefly’s mission is inclusive of all rare neurodegenerative genetic diseases that affect children and have no cure. Our long-term strategy has always been to use our learnings from our initial work for NPC, and where our work can be translated across other rare neurodegenerative genetic diseases, we will be prepared to focus on those diseases as appropriate.