Pharmacologic Treatment Assigned for Niemann Pick Type C1 Disease Partly Changes Behavioral Traits in Wild-Type Mice.

Niemann-Pick Type C1 (NPC1) is an autosomal recessive inherited disorder characterized by accumulation of cholesterol and glycosphingolipids. Previously, we demonstrated that BALB/c-npc1 mice treated with miglustat, cyclodextrin and allopregnanolone generally performed better than untreated animals. Unexpectedly, they also seemed to accomplish motor tests better than their sham-treated wild-type littermates.