NCT02623699 - Tofersen vs Placebo & Amyotrophic Lateral Sclerosis (ALS)

Primary outcome measures:
Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Change from Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score at Week 28
others

In adults with ALS due to SOD1 mutations, CSF SOD1 concentrations decreased at the highest concentration of tofersen administered intrathecally over a period of 12 weeks. CSF pleocytosis occurred in some participants receiving tofersen. Lumbar puncture–related adverse events were observed in most participants
Miller et al. 2020 NEJM 383: 109-119
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