Margarida Maia, PhD, science writer —

Scientists may have discovered promising treatments for Rett syndrome in two small molecules, called KW-2449 and VPA, each seen to ease nerve cell deficits — thereby potentially improving the flow of information between neurons — in a lab model of the disorder. “These findings suggest that KW-2449 and VPA…

NGN-401, a new gene therapy in the pipeline from Neurogene, extended the lifespan and reduced the disease burden in mice with a form of Rett syndrome, the company announced. Researchers also observed that the therapy was safe when delivered to mice or early in the life of…

A mosaic mutation — one that is present in only some body cells — causing Rett syndrome was found in a 2-year-old boy in India, a report says. According to researchers, this is the first time this specific mutation has been reported in a male patient. The report, “…

The loss of MECP2 protein — defective in most cases of Rett syndrome — in the lungs may result in breathing problems, according to a mouse study. The findings suggest that this may due to altered amounts of fats in the lungs, and could have important implications for treating…

The Rett Syndrome Research Trust (RSRT) has given two researchers $1.1 million to collaborate on the development of a therapy capable of switching on a healthy, backup copy of MECP2 — the gene mutated in most cases of Rett syndrome. The collaboration involves Antonio Bedalov, MD, PhD, a…