In line with the notion that chodl is a key factor downstream of the SMN pathway and that synapse loss precedes neuronal death in SMA (Bäumer et al., 2009; Zhang et al., 2008), we reasoned that compounds that rescue synaptic defects in chodl mutants might also ameliorate defects in SMA models. Here, SMN1 is linked to proximal spinal muscular atrophy.