Because of its ability to influence TDP-43-associated toxicity20,25,26, ataxin-2 has emerged as a potentially broadly applicable target for ALS-FTD, as TDP-43 pathology is observed in ~97% of ALS cases1,4,5 and ~45% FTD occurrences1,2,4. The gene discussed is TARDBP; the disease is frontotemporal dementia.