Su et al. (2014) further designed and screened small molecules targeting r(G4C2)n to inhibit repeat-associated non-ATG translation and the formation of RNA foci. The findings from their work suggest that small molecules targeting r(G4C2)n hold promise as a potential therapeutic approach for C9orf72-linked ALS/FTD (Su et al., 2014). The gene discussed is C9orf72; the disease is amyotrophic lateral sclerosis.