Niemann-Pick disease primarily affects children, although it can occur at any age. Types A, B and C all affect the body's ability to metabolize fat (cholesterol and lipids) within cells. When cholesterol and other lipids accumulate in large amounts within cells, they cause cell dysfunction and ultimately cell death. Niemann-Pick disease can affect the brain, nerves, liver, spleen and bone marrow and, in severe cases, the lungs. Progressive deterioration of the brain, nerves and organs cause the main symptoms. Niemann-Pick is an inherited disease with no known cure, but research continues.
April 02, 2014
- Aster JC, et al. Hematopathology. Philadelphia, Pa.: Saunders Elsevier; 2013. https://www.clinicalkey.com. Accessed Oct. 15, 2013.
- Cruse RP. Overview of Niemann-Pick disease. http://www.uptodate.com/home. Accessed Oct. 16, 2013.
- Lichtman MA, et al. Williams Hematology. 8th ed. New York, N.Y.: The McGraw-Hill Companies; 2010. http://www.accessmedicine.com/resourceTOC.aspx?resourceID=69. Accessed Oct. 16, 2013.
- Niemann-Pick disease overview — Types A, B and C. National Niemann-Pick Disease Foundation. http://www.nnpdf.org/npdisease_01.html. Accessed Oct. 16, 2013.
- Treatment options for Niemann-Pick disease. National Niemann-Pick Disease Foundation. http://www.nnpdf.org/npdisease_06.html. Accessed Oct. 16, 2013.