The NCT04768972 trial is now evaluating ION363 in ALS patients with FUS mutations, with early data suggesting dramatic effects, particularly in younger patients, with some showing improvements in ALSFRS-R scores [36].Therefore, proper classification of the pathogenetic variants associated with ALS and a study to understand the harmful molecular mechanisms causing the disease are fundamental steps to develop new specific therapeutic strategies for this fatal disease. The gene discussed is FUS; the disease is amyotrophic lateral sclerosis.