Niemann-Pick disease type C (NPC)

Niemann-Pick disease type C (NPC) is a rare, genetic disease that affects the body’s ability to metabolize fat within cells. It is in a group of disorders called lysosomal storage diseases. In NPC, cholesterol and other fats build up and affect the liver, spleen, lungs or brain.

NPC can occur at any age but primarily affects children. The disease has no known cure, and most children with NPC do not live to adulthood.

The latest on our research

We are currently recruiting families to help make this NPC research database as strong as possible, in partnership with our four foundation partners. We hope to fuel multiple future research projects and studies, and will keep participants updated on our progress.