This highlights the urgent need to acquire and publish more comprehensive human postmortem data on brain and spinal cord distribution of therapeutic ASOs, e.g., the gapmers used in the recent trials in Huntington’s disease (GENERATION HD1 Trial, NCT03761849; Tabrizi et al.61)and SOD1 ALS (VALOR trial, NCT02623699; Miller et al.15). The gene discussed is SOD1; the disease is amyotrophic lateral sclerosis.