The approach, which involves targeting a modifier gene that does not directly cause ALS and reducing Ataxin-2 levels, represents an original therapy for TDP-43 proteinopathy: this type of approach will likely be essential for treating sporadic ALS because of the cellular role of TDP-43, which is present in nearly all ALS patients. The gene discussed is ATXN2; the disease is amyotrophic lateral sclerosis.