Summary

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• This summary is based on the review of one randomized controlled trial(s). ^[1]
• In adults with amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations, the change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score from baseline to week 28 was -6.98 for the tofersen group and -8.14 for the placebo group, resulting in a difference of 1.2 points (95% CI, -3.2 to 5.5; P = 0.97), indicating no significant difference in clinical function between the two groups.
• Secondary endpoints demonstrated significant reductions in concentrations of SOD1 in cerebrospinal fluid and neurofilament light chains in plasma for the tofersen group compared to placebo; however, no significant differences were noted in other secondary clinical endpoints, including slow vital capacity and handheld dynamometry.
• A subgroup analysis indicated that the change in ALSFRS-R score was slightly more favorable in participants predicted to have faster-progressing disease receiving tofersen compared to placebo, although the difference was not statistically significant.
• In adults with amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations, lumbar puncture-related adverse events were common among participants receiving tofersen, with neurologic serious adverse events occurring in 7% of the tofersen group.
• No serious adverse events were observed in the placebo group, and specific details regarding the nature of the neurologic serious adverse events in the tofersen group were not provided.
• The study focused on adults with amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations, with a subgroup analysis of participants predicted to have faster-progressing disease; the change in ALS Functional Rating Scale-Revised (ALSFRS-R) score at week 28 was -6.98 for the tofersen group and -8.14 for placebo, showing no significant difference (1.2 points difference, 95% CI, -3.2 to 5.5; P = 0.97).