Researchers at the Wyss Institute leveraged an AI-enabled drug prediction pipeline to identify vorinostat, an HDAC inhibitor approved for lymphoma, as a promising disease-modifying therapy for Rett syndrome, a rare neurodevelopmental disorder. The approach used gene network analysis and preclinical models including Xenopus tadpoles to demonstrate efficacy across neuronal and systemic symptoms. This target-agnostic framework represents a novel paradigm for addressing complex multisystem diseases.