Furthermore, in humans, a phase 1–2 ascending dose trial evaluating the effect of Tofersen (an ASO that mediates SOD1 degradation) in SOD1 ALS, exploratory results on 50 SOD1 mutated patients showed a slowing in the decrease of ALSFRS-R, slow vital capacity and handheld dynamometry scores during time, a matched decrease of SOD1 protein in the CSF and of pNfH and NfL in plasma and CSF, especially in the group treated with the highest dose (100 mg) and in fast progressors, from baseline to day 85 and to a lesser extent also later on [103]. This evidence concerns the gene SOD1 and amyotrophic lateral sclerosis.