Alterations of NPC components triggered by the impaired export and nuclear accumulation of CHMP7, a critical mediator of NPC, have been shown in Induced Pluripotent Stem Cell-derived ALS motoneurons, to initiate the pathological cascade leading to the mis-localization of TAR DNA binding protein 43 (TDP-43), the hallmark of genetic and sporadic forms of ALS and related neurodegenerative disorders [55]. Here, NPC1 is linked to amyotrophic lateral sclerosis.