Treating Niemann-Pick Disease Type C

Niemann-Pick disease (NPC) is an inherited metabolic disorder where lipids are not able to be metabolized adequately and hence build up and cause damage in various parts of the body such as the spleen, liver, lungs, bone marrow and brain. The different types of the disease are due to different genetic mutations.

There are four most commonly recognized forms of the disease: Types A, B, C, and D. Types A and B are also called Type I. Types C and D are also known as Type II.

Type C is a juvenile or subacute form of the condition which usually starts during childhood and survival into adulthood is possible.

A new treatment for Niemann-Pick disease type C, a progressive neurological condition that affects young people, shows promise in relieving symptoms and improving quality of life.