Primary treatment strategies for SMA aim at boosting SMN protein levels mainly through pharmacologic agent-induced transcriptional activation or splicing pattern alteration of the SMN2 gene, enhancement of SMN2 exon 7 inclusion via antisense oligonucleotides (ASOs) or replacement of mutant SMN genes with functional copies by means of gene therapy vectors. The gene discussed is SMN1; the disease is proximal spinal muscular atrophy.