Niemann-Pick type C (NP-C) disease is a rare, fatal, degenerative, genetic condition primarily affecting children and teenagers, but which can strike at any age. There are approximately 900 known NP-C patients worldwide. The neurological symptoms of NP-C disease are caused by the accumulation of glycosphingolipids in neurons, secondary to abnormal lipid trafficking. NP-C disease is relentlessly progressive. Neurological deterioration is a key feature of the disease and can manifest itself as clumsy body movements, balance problems, slow and slurred speech, swallowing difficulty, eye movement problems, and seizures. Intellectual decline is common. In the final stages of the disease, the child or young adult is frequently bedridden, has little muscle control, and is intellectually impaired.