Steve Bryson, PhD, science writer —

The use of microRNA molecules that targeted a protein receptor important for nerve cell growth led to improved behaviors and motor function in a Rett syndrome mouse model, a study shows. These molecules were carried by tiny vesicles called exosomes, which were derived from human urinary stem cells. The…

A study suggests lower intake of dietary protein is associated with more severe growth deficits in Taiwanese children with Rett syndrome. While dystonia — uncontrolled movements due to muscle contractions — increased with age, it did not affect growth, the scientists…

People with Rett syndrome who grind their teeth during the day had a higher incidence of dental treatment, particularly restorations, than those who did not, an international database study reported.  The findings also showed that patients fed orally had more dental…

Due to the unprecedented challenges caused by the ongoing COVID-19 pandemic, GW Pharmaceutics has stopped the ARCH Phase 3 clinical trial evaluating the oral cannabidiol (CBD) solution Epidiolex in children and adolescents with Rett syndrome. The decision was made after evaluating the progress of…

Problems with skilled learning tasks that involve motor function and making sense of sounds — known to be affected by Rett syndrome — were seen in rat model of the disease and consistent with these deficits in patients, a study reported. These findings could support work into…

Addressing changes in the adenosine system, essential for nerve cell function in the brain, may be a therapeutic strategy in Rett syndrome, a study in mice reported. The study, “Impairment of adenosinergic system in Rett…

To better understand X-linked disorders such as Rett syndrome, researchers discovered an enzyme called DCP1A that acts as a molecular switch to inactivate only one X chromosome, a process essential for normal  development in females. The results were reported in the study, “…

Researchers at the Salk Institute for Biological Studies have mapped how DNA methylation — a process that controls gene expression — changes over time before and after birth in mice to better understand the underlying mechanisms of developmental disorders such as Rett syndrome. …

Treatment with sarizotan failed to reduce apnea episodes (breathing stops) while awake compared to a placebo, failing to achieve the primary goal of the STARS Phase 2/3 clinical trial in people with Rett syndrome and breathing difficulties. …

Newron Pharmaceuticals has received the final minutes from a meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal STARS Phase 2/3 clinical trial investigating sarizotan as a potential treatment for people with Rett syndrome…