Niemann Pick Type C is an inherited progressive and fatal neurodegenerative disorder classified as a lysosomal storage disease. Cells are unable to receive proper instructions to recycle cholesterol and lipids resulting in a devastating accumulation. NPC is often referred to as childhood Alzheimer disease due to parallels with disease progression. The severity of the mutation dictates when the disease will onset. It can be as early as infancy and as late as young adulthood, with most cases presenting during the elementary school years as developmental delay or decline in ability. Later onset is often initially mistaken for dementia or psychiatric disorders. Seizures are common. Patients eventually suffer complete neurological decline and death is often the result of respiratory complications.

At the School of Veterinary Medicine, we have been working to find better treatments for this disease. With our collaborators, we have never been closer to finding a cure for this disease. Last year, support from this Ride helped fund a member of our lab whose job is focused on finding effective therapies.
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