ATXN2 and amyotrophic lateral sclerosis: In the transgenic TDP43 ALS murine model, anti-ATXN2 ASOs rescued the phenotype and prolonged survival, particularly when given early.23A phase 1 clinical trial is ongoing to assess the safety and efficacy of IT anti-ATXN2 ASOs in patients with sporadic and ATXN2-related ALS.21