Finally, the ubiquitin-like modifier activating enzyme Uba1 (Groen and Gillingwater, 2015) and its downstream effectors [including the Wnt signaling effector β-catenin (Ctnnb1)], have been identified as major targets acting downstream of SMN to regulate neuromuscular and systemic pathology in SMA. Here, UBA1 is linked to proximal spinal muscular atrophy.