FIGURE 1.

DS and DR methods as powerful approaches in SMA therapeutic research. The two approaches, eventually in combination, can pave the way for rapid identification of drugs for novel SMA treatments. The compounds (present in large screening libraries) can be tested for primary outcomes (continuous arrow, in the middle) firstly on “simplified” SMA models (cell cultures and/or invertebrates models), and then on SMA mice and/or patient-derived iPSCs (differentiated in neurons, MNs and muscle cells, eventually cocultured) to achieve secondary outcomes. In some cases (dotted arrow, in the middle) the hit compounds can be directly tested on murine models and iPSCs. Created with BioRender software. DR, drug repositioning; DS, drug screening; MN, motor neuron; SMA, spinal muscular atrophy.