SMN2 and proximal spinal muscular atrophy: We identified a SMN2 splicing small-molecule modulator, termed TEC-1 (2-(4,6-dimethylpyrazolo[1,5-a]pyrazin-2-yl)-6-(4-methylpiperazin-1-yl)quinazolin-4(3H)-one)20, that shows promise as a clinical candidate for the tolerable long-term treatment of SMA.