Drug screen and machine learning predict neuroprotective agents in a preclinical human model of childhood dementia

Nature Communications, Published online: 20 August 2026; doi:10.1038/s41467-026-76837-1 Here authors show patient-derived neural models of childhood dementia exhibit key disease features. A drug screening platform integrating machine learning, imaging, transcriptomics and electrophysiology, identifies repurposed compounds that mitigate adverse effects.