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Xanthomatosis, Cerebrotendinous
An autosomal recessive lipid storage disorder due to mutation of the gene CYP27A1 encoding a CHOLESTANETRIOL 26-MONOOXYGENASE. It is characterized by large deposits of CHOLESTEROL and CHOLESTANOL in various tissues resulting in xanthomatous swelling of tendons, early CATARACT, and progressive neurological symptoms.
Drugs by status
Phase 2 trials (2)
Organizations
Organization Involved with Phase 2 Indications (2)
Organization Involved with Other Experimental Indications (1)