NPC1 and Niemann-Pick disease type C: However, in contrast to the functional rescue achieved with the oxysterol chaperones, itraconazole was much less efficient in reducing cholesterol accumulation in patient-derived fibroblasts, suggesting that itraconazole itself may not be suitable for treatment of Niemann-Pick disease type C. Still, considering its high potency in correcting the localization defect of the NPC1 mutant, it could be a promising lead compound for the development of non-steroidal chaperone drugs through further medicinal chemistry optimization.