Our results demonstrate that VPAC2 receptor agonist BAY 55-9837 increases SMN protein levels and attenuates disease progression in two distinct severe SMA mouse models (Figure 5) providing a proof of concept and support for other VPAC2 agonists/p38 activating compounds to be tested as effective SMA therapies. The gene discussed is VIPR2; the disease is proximal spinal muscular atrophy.