Feasibility of the approach is supported by FDA approval of Spinraza for the treatment of spinal muscular atrophy (83, 84), the recently completed clinical trial with an ASO for Huntington’s (Htt) disease (46) and ongoing trials for an SNCA-targeted ASO for synucleinopathy (NCT04165486), a SOD1-targeted ASO for amyotrophic lateral sclerosis (NCT02623699), a C9ORF72-targeted ASO for ALS (NCT03626012), a LRRK2-targeted ASO for PD (NCT03976349), and a MAPT-targeted ASO therapy for Alzheimer’s disease (NCT03186989). This evidence concerns the gene SOD1 and early-onset autosomal dominant Alzheimer disease.