The data herein suggest that treatment with HSP-amplifying compounds may be a promising therapeutic strategy for improving white matter thickness and counteracting hypomyelination, which is associated with the clinical manifestations of ataxia and impaired ocular-motor functioning in NPC, and therefore provide further mechanistic detail on the disease-modifying effects of HSP amplification by this class of compounds. This evidence concerns the gene HSP90B2P and cerebellar ataxia.