Therapeutic intervention targeting repeat-associated non-ATG (RAN) translation demonstrates efficacy in ameliorating symptoms of amyotrophic lateral sclerosis and frontotemporal dementia while leaving the underlying RNA repeat expansions intact. This mechanism-specific approach appears to disrupt the production of toxic dipeptide repeat proteins that accumulate in affected neurons without requiring correction of the genetic root cause. The strategy represents a significant departure from gene therapy approaches that attempt to reduce or eliminate the expanded RNA repeats themselves. For individuals with C9orf72-linked ALS and FTD, this could offer a more feasible therapeutic pathway since it bypasses the technical challenges of delivering genetic corrections to motor neurons and brain tissue. The selectivity for RAN translation over normal protein synthesis suggests a potentially favorable safety profile, though long-term neurological effects remain to be established. This finding builds on growing evidence that dipeptide repeat proteins, rather than RNA toxicity alone, drive much of the neurodegeneration in these conditions. The approach may be particularly valuable given that current ALS treatments provide only modest symptomatic relief, and no disease-modifying therapies exist for FTD.