ATXN2 and amyotrophic lateral sclerosis: These promising preclinical studies led to the clinical trial on the ATXN2-targeting ASO, BIIB105 to reduce ataxin-2 in ALS patients with or without intermediate length ATXN2 CAG repeats (https://alsnewstoday.com/news/biib105-trial-enrolling-als-with-without-atxn2-mutations/, accessed on 5 May 2024).