Cerebrotendinous xanthomatosis is an inherited autosomal recessive lipid storage disease caused by a 27-hydroxylase enzyme
deficiency, characterised clinically by tendon xanthomas, premature cataracts, chronic diarrhoea and progressive neurologic
dysfunction. The disease is very uncommon and there are very few pathological descriptions. We report a 52-year-old male who
presented with a neuropsychiatric disorder and cognitive decline. Despite treatment the patient developed optic atrophy, parkinsonism
and dementia and died. The autopsy revealed a nonspecific brain and cerebellar atrophy.Under microscopic examination, lipid
crystal clefts, neuronal loss, demyelination, reactive astrocytosis and perivascular macrophages were found. These findings
suggest the limited reversibility of the disease, and its poor prognosis, specially if treatment is not started early.
Key words cerebrotendinous xanthomatosis - 27-hydroxylase - CYP27A1 - necropsy