Summary

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• This summary is based on the review of one randomized controlled trial. ^[1]
• N-Acetyl-l-Leucine (NALL) in Niemann–Pick Disease Type C: In patients with genetically confirmed Niemann–Pick disease type C, treatment with NALL resulted in a significant mean change in the Scale for the Assessment and Rating of Ataxia (SARA) total score of -1.97 ± 2.43 points after 12 weeks, compared to -0.60 ± 2.39 points in the placebo group. The least-squares mean difference between NALL and placebo was -1.28 points (95% confidence interval (CI), -1.91 to -0.65; P < 0.001).
• Secondary outcomes, including the Clinical Global Impression of Improvement, Spinocerebellar Ataxia Functional Index, and Modified Disability Rating Scale, generally supported the primary findings, although these were not adjusted for multiple comparisons.
• The incidence of adverse events was similar between NALL and placebo, with no treatment-related serious adverse events reported.
• There were no significant safety concerns or adverse effects reported in the reviewed studies.
• There is no population types or subgroups information available in the reviewed studies.