Niemann-Pick Disease

Niemann-Pick disease is a group of rare conditions passed down in families. The conditions affect the body’s ability to break down and use fats, such as cholesterol and lipids, inside cells. Because of the buildup of fats, these cells don’t work as they should and, over time, the cells die. Niemann-Pick disease can affect the brain, nerves, liver, spleen and bone marrow. Sometimes it can affect the lungs.

Symptoms of Niemann-Pick disease relate to worsening function of the nerves, brain and other organs over time.

Niemann-Pick disease can happen at different ages but mainly affects children. The condition has no known cure and is sometimes fatal. Treatment is focused on helping people live with their symptoms.