Summary

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• This summary is based on the review of two randomized controlled trial(s). ^[1-2]
• Functional Decline (Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) Score): Sodium phenylbutyrate-taurursodiol (PB-TURSO) slowed the decline in the ALSFRS-R score over 24 weeks compared to placebo, with a mean rate of change of -1.24 points per month for the active drug versus -1.66 points per month for placebo. The difference was 0.42 points per month (95% confidence interval (CI), 0.03 to 0.81; P = 0.03).
• Survival: Median overall survival was 25.0 months for participants randomized to PB-TURSO, compared to 18.5 months for placebo, reflecting a 6.5-month longer survival for the PB-TURSO group (hazard ratio, 0.56; 95% CI, 0.34-0.92; P = 0.023).
• Secondary Outcomes: There were no significant differences between PB-TURSO and placebo in secondary outcomes, including isometric muscle strength, plasma phosphorylated neurofilament levels, slow vital capacity, and time to death or hospitalization.
• Adverse Events: Adverse events associated with PB-TURSO were predominantly gastrointestinal in nature.
• There is no information on significant safety concerns or adverse effects in specific population types or subgroups in the reviewed documents.
• There is no population types or subgroups information available in the reviewed documents.