As shown recently [32–35], similar to Nusinersen treatment for SMA (spinal muscular atrophy), NVP-13 showed effective brain concentrations between 2 and 100 μg NVP-13/g tissue, regulating TGFβRII expression in CNS subregions SC, SVZ (subventricular zone), and HIP (hippocampus) (Fig. 2C, D). Here, SMN1 is linked to proximal spinal muscular atrophy.