SOD1 and amyotrophic lateral sclerosis: In addition, an ASO that activates RNase-H-mediated mRNA degradation has exhibited promising effects on patients with SOD1-related ALS (ALS1) and is now in a phase III study (https://clinicaltrials.gov/ct2/show/NCT02623699?term=Tofersen&draw=2&rank=1), supporting the efficacy of ASOs in treating ALS caused by a gain-of-toxicity mutation.