Indeed, although in the last years different translational approaches, both SMN-dependent (e.g., RNA-based modulation of SMN2) and SMN-independent (e.g., neurotrophic factors, stem cells), have been adopted to develop therapeutics for SMA patients (Lorson et al., 2010; Boido and Vercelli, 2016), the molecular pathways involved in neurodegeneration remain unknown. The gene discussed is SMN1; the disease is proximal spinal muscular atrophy.