Genetic and Rare Diseases Information Center (GARD)


Other names people use for this condition
  • CTX
  • Cerebral cholesterinosis

Cerebrotendinous xanthomatosis
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Cerebrotendinous xanthomatosis (CTX) is a type of lipid storage disease. It is caused by mutations in the CYP27A1 gene.[1][2] Symptoms of cerebrotendinous xanthomatosis include infantile-onset diarrhea, cataract, tendon xanthomas, and progressive neurologic dysfunction.[2] Treatment may involve chenodeoxycholic acid (CDCA), inhibitors of HMG-CoA reductase, and cataract extraction.[2]

References
  1. NINDS Lipid Storage Diseases Information Page. National Institute of Neurological Disorders and Stroke. 2008 Available at: http://www.ninds.nih.gov/disorders/lipid_storage_diseases/lipid_storage_diseases.htm. Accessed June 8, 2008.
  2. Federico A, Dotti MT, Gallus GN. Cerebrotendinous Xanthomatosis. GeneReviews. 2008 Available at: http://www.ncbi.nlm.nih.gov/bookshelf/br.fcgi?book=gene&part=ctx. Accessed June 08, 2008.

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