Cerebrotendinous xanthomatosis


Disease modifying treatment available:
Time critical diagnosis and management:
Lateralising:


Aetiology

Disorder Clinical features Associated features Investigations Disease modifying treatment available Time critical
Dementia Cerebrotendinous xanthomatosis
Xanthomas

Associated genes

Gene
CYP27A1 Search ClinVar Search ClinGen



Associated Disorders





Clinical features


Cerebrotendinous xanthomatosis may cause the following:





Investigations


Due to the nature of the biochemical defect, the cholestanol concentration in plasma or serum (derived from blood) is high, while the plasma cholesterol concentration is normal to low. In addition, the concentration of bile acid pathway intermediates is elevated, and the concentration of bile alcohols in plasma, bile and urine is increased. Bile alcohols are formed in an alternative pathway present in CTX that generates some cholic acid. Due to the nature of the biochemical defect in CTX there is little or no formation of chenodeoxycholic acid.


Laboratory Investigation Result