Less is known about mitochondrial transport in general with respect to NP-C, whereas a contribution of reduced transport of organelles to the pathophysiology has been described for several other neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS) [8] or spinocerebellar ataxia 20 (SCAR20) [9]. Here, NPC1 is linked to neurodegenerative disease.