SpinrazaTM is an ASO with the ability to bind to SMN2 pre-mRNA and hinder the attachment of the heterogeneous nuclear ribonucleoprotein (hnRNP); thereby exposing the splice site responsible for inclusion of exon 7 to produce a full-length, functional SMN2 mature mRNA, which ameliorates SMA disease progression by restoring motor neuron function [107,108]. Here, SMN2 is linked to proximal spinal muscular atrophy.