Based on a high-throughput screen of 3765 small-molecule derivatives of Pimozide, Bose et al. [104] identified a small molecule, TRVA242, which significantly rescued the locomotor, motoneuron and NMJ deficiency in ALS models of C. elegans with TDP-43 mutant and zebrafish with multiple mutants (TDP-43, SOD1, and C9orf72). Here, TARDBP is linked to amyotrophic lateral sclerosis.