Niemann-Pick Type C Disease (NP-C) is a genetic pediatric neurodegenerative disorder causing progressive deterioration of the nervous system. It usually starts to affect children of school age (4-7 years old) by interfering with their ability to metabolize cholesterol within the cell. Consequently, large amounts of cholesternpc1ol accumulate within the liver, spleen, and brain. This metabolic disorder leads to a series of neurological problems that are ultimately FATAL.
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