Efficacy in familial ALS is supported by the data in cellular assays as well as in transgenic C. elegans (worms), D. melanogaster (flies) and mice bearing a diversity of ALS-causing transgenes (TDP-43 A315T, c9orf72 repeat expansion, and SODG93A, respectively (Figures 1–4). This evidence concerns the gene C9orf72 and amyotrophic lateral sclerosis.