Some experimental RNA-based therapies, including small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs), have already reached clinical trials, such as tofersen (196), an ASO targeting SOD1 mutations in amyotrophic lateral sclerosis (ALS), highlighting the translational potential of ncRNA-based interventions. Here, SOD1 is linked to amyotrophic lateral sclerosis.