Tofersen Fails to Slow SOD1-ALS in VALOR Trial, But Hopeful Trends Seen

Tofersen Fails to Slow SOD1-ALS in VALOR Trial, But Hopeful Trends Seen

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Tofersen Fails to Slow SOD1-ALS in VALOR Trial, But Hopeful Trends Seen

Tofersen, an experimental treatment for amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, failed to significantly slow the rate of disease progression relative to a placebo in a Phase 3 trial. These top-line findings mean that the VALOR study (NCT02623699), which is testing tofersen in 108 adults with SOD1-ALS, did not meet its primary goal. Trends favoring tofersen were seen across other measures, however, including assessments of muscle strength, life quality, and lung…

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