ASOs have been given intrathecally to patients with ALS; treatment has been shown to be well tolerated and result in significantly lowered SOD1 levels in the CSF.195 A phase I/II trial with torfersen ASO in 50 patients with SOD1 mutations not only revealed a good tolerance profile, but also suggested that the clinical impact of this therapy is worth exploring in a phase III trial.196 In October 2021, results of the tofersen phase III study (VALOR) were presented at the American Neurological Association annual meeting (https://investors.biogen.com/). Here, SOD1 is linked to amyotrophic lateral sclerosis.