This study reports the generation of induced pluripotent stem (iPS) cells from skin fibroblast samples of a child with spinal muscular atrophy (SMA), a genetic disorder causing infant mortality due to selective loss of lower motor neurons. The iPS cells expanded robustly in culture, maintained the disease genotype, and generated motor neurons that showed selective deficits compared to those from the child's unaffected mother. This is the first study to demonstrate that human iPS cells can model the specific pathology of SMA, making them a promising resource for studying disease mechanisms, screening novel drug compounds, and developing new therapies. The iPS-SMA cells responded to compounds known to increase SMN protein, suggesting their potential use in drug screening. The study also highlights the importance of using patient-specific iPS cells to better understand and treat genetically inherited diseases.This study reports the generation of induced pluripotent stem (iPS) cells from skin fibroblast samples of a child with spinal muscular atrophy (SMA), a genetic disorder causing infant mortality due to selective loss of lower motor neurons. The iPS cells expanded robustly in culture, maintained the disease genotype, and generated motor neurons that showed selective deficits compared to those from the child's unaffected mother. This is the first study to demonstrate that human iPS cells can model the specific pathology of SMA, making them a promising resource for studying disease mechanisms, screening novel drug compounds, and developing new therapies. The iPS-SMA cells responded to compounds known to increase SMN protein, suggesting their potential use in drug screening. The study also highlights the importance of using patient-specific iPS cells to better understand and treat genetically inherited diseases.