The two very recent accelerated approvals by the Food and Drug Administration (FDA) of Eteplirsen, a phosphorodiamidate morpholino AON that modulates splicing in the Dystrophin gene to treat patients with Duchenne muscular dystrophy,47, 48 and Nusinersen, an MOE AON that modulates splicing in the SMN2 gene to treat patients with spinal muscular atrophy,49, 50 are a huge step forward in facilitating AONs as therapeutic strategies in genetic diseases and will undoubtedly promote similar development in other neuromuscular diseases. The gene discussed is SMN2; the disease is proximal spinal muscular atrophy.