Described is a method for treating an individual having a neurological
disorder with an associated mutation or mutations in a gene encoding a
lysosomal enzyme. Specifically, the individual is administered a specific
pharmacological chaperone for the lysosomal enzyme which increases
trafficking of the protein from the ER to the lysosome in neural cells,
with or without concomitantly increasing enzyme activity in neural cells.
Restoration of trafficking relieves cell stress and other toxicities
associated with accumulation of mutant proteins. Restoration of enzyme
activity relieves substrate accumulation and pathologies associated with
lipid accumulation. In a specific embodiment, the neurological disorder
is Parkinson's disease or parkinsonism which is associated with mutations
in glucocerebrosidase.