Tofersen does not improve ALS symptoms in patients with SOD1 mutations | 2 Minute Medicine

1. In patients with amyotrophic lateral sclerosis (ALS) and superoxide dismutase 1 (SOD1) mutations, tofersen does not result in significant functional improvement as compared to placebo. 2. In comparison to the placebo, tofersen significantly reduced SOD1 concentrations in the cerebrospinal fluid (CSF) and plasma of persons with faster progressing SOD1 ALS. Evidence Rating Level: 1

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