The only therapy that is presently approved for SMA is nusinersen (SPINRAZA), which is an antisense-oligonucleotide (ASO) that binds to the SMN2 pre-mRNA downstream of exon 7, promoting its incorporation into the mRNA and leading to the translation of a fully functional SMN protein [6]. The gene discussed is SMN1; the disease is proximal spinal muscular atrophy.