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The primary outcome, assessed by measuring the change in the Scale for the Assessment and Rating of Ataxia (SARA) scores from baseline to 12\u2009weeks post\u2010treatment, is central to evaluating treatment efficacy. Secondary outcomes\u00a0included changes in the International Cooperative Ataxia Rating Scale (ICARS) score, Barthel Index of Activities of Daily Living (ADL), and plasma and cerebrospinal fluid (CSF) GABA levels. Safety was assessed in all treated patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 48 patients with SCA3 were enrolled in this study. After 12\u2009weeks, data from 43 patients were included in the efficacy analysis (intention\u2010to\u2010treat analysis). The least\u2010squares mean change in the SARA score from baseline to 12\u2009weeks post\u2010treatment was \u22123.80 (standard error [SE], 0.39; 95% confidence interval [CI], \u22124.58 to \u22123.02) in the high\u2010dose GM1 group, 0.34 (SE, 0.40; 95% CI, \u22120.46 to 1.13) in the low\u2010dose GM1 group, and 0.73 (SE, 0.40; 95% CI, \u22120.07 to 1.52) in the placebo group, respectively. Secondary outcomes showed improvements in the ICARS score, Barthel Index of ADL, and plasma and CSF GABA levels in the high\u2010dose GM1 group compared to the low\u2010dose GM1 and placebo groups. All treatments were well\u2010tolerated and safe.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>High\u2010dose GM1 treatment significantly ameliorated ataxic symptoms in patients with SCA3. \u00a9 2024 International Parkinson and Movement Disorder Society.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mds.30050","type":"journal-article","created":{"date-parts":[[2024,11,7]],"date-time":"2024-11-07T08:55:02Z","timestamp":1730969702000},"page":"57-66","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Potential Disease\u2010Modifying Effects of Ganglioside <scp>GM1<\/scp> Pulse Treatment on Spinocerebellar Ataxia Type 3, a Parallel\u2010Group, Double\u2010Blind, Randomized, Controlled 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