Combined therapy with cyclodextrin/allopregnanolone and miglustat improves motor but not cognitive functions in Niemann–Pick Type C1 mice.

Highlights: [•] Standard behavioral tests were successfully applied to NPC1−/− mutant mice. [•] The mutant mice revealed defective motor performance and cognitive impairment. [•] The motor impairment was in agreement with Purkinje cell degeneration in the cerebellum. [•] The motor performance improved significantly upon combined therapy. [•] The impaired cognitive function, however, did not benefit from the treatment. [Copyright &y& Elsevier]