These various approaches are now in clinical trials, with promising outcomes that support the predictive validity of the animal models (Finkel et al. 2017; Mendell et al. 2017; Mercuri et al. 2018), and nusinersen, the ASO promoting SMN2 splicing, was recently approved by the US Food and Drug Administration and the European Medicines Agency as the first treatment for SMA. The gene discussed is SMN2; the disease is proximal spinal muscular atrophy.