Intravenous esketamine in pediatric Rett syndrome: An open-label, early phase 1 pilot study.
Molecular therapy. Methods & clinical development March 13, 2025 Huiping Li, Shu Liu, Caimei Lin et al. 5 citations
Intravenous esketamine, an NMDAR antagonist, was tested in a small pilot study of three girls with Rett syndrome aged 5-10 years. The treatment was generally well tolerated, with mild, self-resolving side effects such as nausea, vomiting, and irritability. Participants showed minimal improvements on behavioral and motor assessment scales, and video electroencephalograms indicated gradual increases in posterior dominant rhythm peak frequency. Individual differences were observed in efficacy measures. The findings suggest potential for esketamine in improving behavioral dysfunction in Rett syndrome, but further research on appropriate dosage forms is needed.