Molecular therapy. Methods & clinical development

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

Rett syndrome (RTT) is a severe neurodevelopmental disorder. N-Methyl-d-aspartate receptor (NMDAR) antagonism has shown therapeutic potential in preclinical RTT models. We performed a pilot study to explore whether intravenous esketamine, an NMDAR antagonist, alleviates the symptoms of pediatric RTT. This was a prospective, single-arm, single-site, open-label, early phase 1 pilot study. Three...