Furthermore, administration of a stabilized form (NT-1654) of the C-terminal 44 kDa fragment of motor neuron-derived agrin, a MuSK activator, enhances NMJ formation and improves motor behavior and survival of a mouse model of spinal muscular atrophy (Boido et al., 2018). The gene discussed is AGRN; the disease is proximal spinal muscular atrophy.