A first randomized-blinded preclinical study in SMA mice testing an antisense oligonucleotide-based combinatorial therapy targeting SMN and Ncald, showed a synergistic amelioration of SMA hallmarks, such as electrophysiological and morphological properties of NMJs and muscles at postnatal day (PND) 21 [30]. Here, SMN1 is linked to proximal spinal muscular atrophy.