If confirmed in other studies with more therapeutic or pathogenic purposes, our data suggest that systemic therapeutic approaches increasing SMN levels also in skeletal muscle may provide additional benefits to SMA patients, and that miR-181a-5p (and/or miR324-5p) modulation might be a potential target for combinatorial treatments in addition to SMN modulation. This evidence concerns the gene SMN2 and proximal spinal muscular atrophy.