News

Marvel Biosciences plans to ask the U.S. Food and Drug Administration (FDA) to grant orphan drug status to its MB-204 treatment candidate for Rett syndrome — with the goal of helping advance the therapy toward clinical studies. This designation is granted by the FDA to incentivize the development…

Computer modeling confirms the weight-based dosing strategy for Daybue (trofinetide) to treat people with Rett syndrome, ages 2 and older. That’s according to two, back-to-back studies published separately in Advances in Therapy. Daybue, from Acadia Pharmaceuticals, is the first therapy approved in the U.S. for people with…

Treatment with NA-921 was found to improve measures of abnormal behavior and overall health in girls and young women with Rett syndrome in a Phase 2/3 clinical trial. Data also suggested the oral therapy is less likely to cause digestive side effects than Daybue (trofinetide), which is marketed…

Dysfunction of an RNA molecule called NEAT1 contributes to nerve cell damage in Rett syndrome, a study suggested, implying that normalizing the activity of this molecule could be a viable treatment strategy for Rett. The study, “NEAT1-mediated regulation of proteostasis and mRNA localization impacts autophagy dysregulation…

A new study aims to raise awareness about Rett syndrome in boys and explore how the disorder affects them and their families. The project is being led by Talia Thompson, PhD, and Timothy Benke, MD, PhD, both professors at the University of Colorado. It’s funded by the International…

Treatment with esketamine — a molecule chemically related to the dissociative anesthetic ketamine — has the potential to ease the symptoms of Rett syndrome, according to researchers in China who…

Treatment with Daybue (trofinetide) in clinical trials helped girls and young women with Rett syndrome to communicate and use their hands better, according to interviews with caregivers of participants in two studies that tested the now-approved therapy. “Caregivers from both trials described a range of improvements, including increased…

Researchers identified a new mutation in the MeCP2 gene in a Chinese boy diagnosed with Rett syndrome, a condition that rarely affects males. The boy exhibited diverse symptoms, including limited movements, breathing and sleep problems, heart and lung defects, and abnormal brain wave activity. “Our discovery of…

Digestive problems, such as constipation and bloating, affect more than 80% of girls and women with Rett syndrome, a study reveals. The most frequent problems included constipation, difficulties with eating and swallowing, and gas and bloating. More than half of the patients showed irritability due to stomach or gut…

Acadia Pharmaceuticals has submitted a marketing authorization application for trofinetide to European Union regulators, seeking the therapy’s approval for adults and children, ages 2 and older, with Rett syndrome. The application to the European Medicines Agency (EMA) is Acadia’s first such submission in Europe, according to a…