Marta Figueiredo, PhD, science writer —

Problems with nerve cell communication and adaptation in the brain’s motor cortex is associated with the severity of motor difficulties in people with Rett syndrome, a study shows. The research — which used a non-invasive method to show such neural deficits in Rett patients for the first time —…

Treatment with insulin-like growth factor 1 (IGF-1) eases symptoms of Rett syndrome by suppressing the activity of FXYD1 — a molecule found in higher-than-normal levels in Rett patients — according to a study in a mouse model of the disorder. Besides supporting the previously reported potential…

The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to trofinetide, a potential oral therapy for people with Rett syndrome. The designation provides priority review to investigational treatments with potential to provide clinically meaningful benefits in serious or life-threatening rare diseases mainly affecting individuals younger…

Restoring the levels of Wnt6, a signaling molecule involved in brain function, eased motor and behavioral difficulties in a mouse model of Rett syndrome, a study shows. These findings added to scientists’ knowledge of the underlying molecular mechanisms of Rett and may help develop new therapies for those…

High-quality speech therapy and more access to augmentative and alternative communication (AAC) tools are needed in schools for girls with Rett syndrome, according to a U.S. survey. The survey’s findings, “Special Education Supports and Services for Rett Syndrome: Parent Perceptions and Satisfaction,” were published in…

The United States Patent and Trademark Office (USPTO) granted a patent to Anavex Life Sciences covering Anavex 2-73 (blarcamesine) as a potential therapy for neurodevelopmental disorders, including Rett syndrome. The patent (No. 10,507,196) is valid until at least 2037 and also covers methods of treatment based on…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to Anavex 2-73 (blarcamesine) for the treatment of Rett syndrome. This designation provides priority review to investigational treatments that have the potential to provide clinically meaningful benefits in serious rare diseases mainly affecting individuals younger than 18. If…

Treatment with Anavex 2-73 was seen to improve motor skills, acoustic responses and visual acuity in a mouse model of Rett syndrome, supporting ongoing Phase 2 studies in patients. Its use also helped to lessen abnormal movements and ease breathing in these mice, its researchers said. The study, “…

Taking coenzyme Q10 (CoQ10) — a natural antioxidant — as an oral dietary supplement can ease oxidative stress associated with symptom severity and Rett syndrome progression, a small Italian study suggests. The study, “Effects of oral administration of common antioxidant supplements on the energy metabolism of red blood cells.

A newly identified mutation in the MECP2 gene — which is linked to Rett syndrome  — was found in two females in Germany who lacked all the common symptoms of the disorder, suggesting these are two cases of atypical Rett syndrome. The report, “Novel MECP2…