Enrichment analysis in mouse phenotypes highlighted several pathways known to be involved in SMA, such as lipid and glucose metabolism (44, 46), as well as muscle fiber morphology and contraction (refs. 45, 47; Figure 10B), providing potential molecular explanations for the improved phenotypes in harmine-treated SMA mice and a similarity to the pathways associated with Pip6a-PMO treatment (Figure 2B). Here, SMN1 is linked to proximal spinal muscular atrophy.