Firefly Fund supporters believe that every child deserves the opportunity to have a life filled with good health so that they can reach their full potential.
Children born with neurodegenerative diseases that go undiagnosed and untreated typically die before the age of 20. The Firefly Fund is working hard to change this grave reality.
Today the Firefly Fund is focused on Niemann Pick Type C (NPC), also referred to as Childhood Alzheimer’s, which is caused by recessive genetic mutations that lead to defective NPC protein. This ultimately leads to progressively debilitating and fatal neurovisceral disease. Currently there are no FDA approved therapies to treat NPC and there is no cure.
With Your Help, We Can Make a Difference
Your contribution will go directly towards the programs of the Firefly Fund:
NPC can be detected in newborns with a simple blood test commonly referred to as newborn screening. The Firefly Fund is leading a nationwide effort to add NPC to newborn screening lists at the state and federal levels. #1dollar1foot. For every dollar donated, one child will receive a screening.
The Firefly Fund supports NPC scientists in conducting innovative research with the goal of finding therapies and ultimately a cure for NPC.
Doctors familiar with treating patients diagnosed with NPC are often far from where families live, which can present a logistical and financial burden for many families. The Firefly Fund supports families who face these challenges by providing assistance for travel, lodging and access to trial medications.
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.