Many aspects of the pathophysiology of the disease remain still controversial or poorly elucidated even in the most common forms of ALS, such as those caused by mutations in C9ORF72 (40%), SOD1 (20%), FUS (1–5%), and TARBDP (1–5%) genes, respectively for ALS-FTD1, ALS1, ALS6 and ALS10 [2,3,4]. The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.