Together with pre-clinical data from the FUS-ALS knock-in mouse models of disease, this first neuropathological study of a single patient with ALS-FUS treated with ION363 provides preliminary evidence in support of FUS silencing as a promising therapeutic strategy to delay or prevent disease onset in pre-symptomatic carriers of ALS-associated FUS mutations and to slow disease progression in patients with ALS-FUS32. This evidence concerns the gene FUS and amyotrophic lateral sclerosis.