The highest dose of the drug administered intrathecally over a period of 12 weeks produced a significant decrease in CSF SOD1 concentrations; importantly, this was accompanied by a decrease of both CSF and plasma concentrations of NFL (and pNFH), representing one of the first examples of the use of neurofilaments as pharmacodynamic biomarkers for an ALS treatment trial (Miller et al., 2020). Here, NEFL is linked to amyotrophic lateral sclerosis.