Patricia Inácio, PhD, science writer —

Mutations in the MECP2 gene, the underlying cause of Rett syndrome, affect  genes that regulate several cellular functions, including inflammation, cellular stress responses, and organization of cellular structures. The study with that finding, “Integrated analysis of human transcriptome data for Rett syndrome finds a network of involved genes,” was published in…

The first patient has been dosed in Anavex Life Sciences’ Phase 2 trial evaluating Anavex 2-73 for the treatment of Rett syndrome. The trial (NCT03758924) assessing the safety, tolerability, and efficacy of Anavex 2-73 in 15 adults (18 and older) with Rett syndrome is going on at three clinical sites in the…

Three-dimensional microbrains built in the lab using stem cells from Rett syndrome patients can become a new model to study the disease, neuroscientists say. This can help identify new therapeutic compounds to treat Rett syndrome and accelerate therapy discovery for other neurodevelopmental disorders. The study, “3-Dimensional Human Cortical Neural…

Patient enrollment is now complete for the pivotal STARS study investigating the safety, tolerability, and effectiveness of sarizotan in Rett syndrome patients with respiratory abnormalities, Newron Pharmaceuticals announced. Top line results from the Phase 2/3 clinical trial are expected in the last quarter of 2019. Sarizotan was developed…