Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are born with it and much fewer boys. It is caused by mutations in the MeCP2 gene on the X chromosome, leading to a spectrum of cognitive and physical impairments, including repetitive hand motions, speech difficulties, and seizures.
AI-guided drug discovery forges a novel therapeutic path for patients with Rett syndrome
