Notably, loss-of-function mutations in the ATP13A2 gene seem causally linked to neurodegenerative diseases, including Kufor-Rakeb syndrome, a rare form of inherited juvenile-early onset Parkinson’s disease [93,94,95,96,97,98,99,100,101], the while enhancement of ATP13A2 function has been proposed as a neuroprotective therapeutic strategy in Parkinson’s disease [100,102,103,104]. This evidence concerns the gene ATP13A2 and Kufor-Rakeb syndrome.