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…
Newron Pharmaceuticals will meet with the U.S. Food and Drug Administration (FDA), which requested a meeting to discuss the company’ statistical plan for releasing data from its pivotal STARS Phase 2/3 study of sarizotan in treating Rett syndrome patients with breathing problems.
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