Three-Dimensional Stem Cell-derived Spinal Cord Model for Investigating Therapeutic Mechanisms of Risdiplam-like Compounds in Spinal Muscular Atrophy (P2-11.001)

To investigate the therapeutic mechanisms of a Risdiplam-like compound in a three-dimensional stem cell-derived spinal cord model for Spinal Muscular Atrophy (SMA) and assess its efficacy in reversing the disease's pathological hallmarks.