However, most mutations are found in only four of them (i.e. C9orf72, TARDBP, FUS and SOD1), which account for almost 75% familial amyotrophic lateral sclerosis and 20% sporadic amyotrophic lateral sclerosis cases.1,4,5 Despite being the most common form of MN disease (MND) in adults, effective treatments for amyotrophic lateral sclerosis, particularly for sporadic forms, remain elusive. This evidence concerns the gene TARDBP and amyotrophic lateral sclerosis.