Mutations in the NPC1 gene account for 95% of NPC patients.80 Degeneration of cerebellar Purkinje neurons is a prominent early feature in the disease progression, which leads to clinical symptoms of motor impairments.81 Very little is known about the cellular death mechanisms leading to neuronal loss in NPC1, and thus the potential efficacy of cell death inhibitors remains unexplored. This evidence concerns the gene NPC1 and nasopharyngeal carcinoma.