Despite the fact that ALS-associated changes detected in peripheral cells from patients might not fully reflect those of motor neurons, we have evaluated the potential neuroprotective role of Tideglusib in a neuronal cell model of induced TDP-43 phosphorylation driven by ethacrynic acid, as well as the efficacy of chronic oral administration of Tideglusib in transgenic TDP-43 (A315T) mice, one of the first experimental models of ALS based on mutations in TDP-43 protein [33]. This evidence concerns the gene TARDBP and amyotrophic lateral sclerosis.