Even though the first large-scale clinical trial (CT) demonstrating clinical efficacy of riluzole to improve survival in ALS patients was conducted in 1990, 3 years prior to the generation of the first SOD1 mouse model (Bensimon et al., 1994), riluzole has subsequently been shown to have similar efficacy in this transgenic model as seen in human ALS trials (Gurney, 1997), thus validating the preclinical model. This evidence concerns the gene SOD1 and amyotrophic lateral sclerosis.