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Firefly Fund
http://fireflyfund.org/
Firefly Fund's 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. Firefly Fund has begun its journey with a focus on Niemann-Pick Type C (NPC).
Your donations fund the research necessary to develop new drug therapies that slow the progression of Niemann-Pick Type C.