A Nature Communications study showed targeted antisense oligonucleotide (ASO) therapy could rescue developmental defects in spinal muscular atrophy (SMA) organoids. The team demonstrated restoration of neuronal phenotypes and improved cellular function in patient-derived three-dimensional models, supporting ASO approaches as viable interventions during neurodevelopmental windows. Authors detailed molecular rescue pathways and suggested the findings could inform timing and dosing strategies for clinical ASO regimens. The organoid results provide a translationally relevant preclinical dataset that may influence trial design and regulatory discussions for pediatric SMA interventions.