Marisa Wexler, MS,  senior science writer—

NGN-401, a gene therapy candidate for Rett syndrome that’s now being tested in a Phase 1/2 clinical trial, was demonstrated to ease symptoms and improve survival in a mouse model of the disease, per new study data. The Neurogene therapy, which uses the company’s proprietary EXACT technology,…

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 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…

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…

A new study sheds light on how MeCP2 protein dysfunction leads to brain abnormalities in Rett syndrome, and provides a framework to develop new treatments like gene therapies. Specifically, researchers conducted a study in adult mice and found the loss of the MeCP2 protein — not problems during development…

Health Canada has approved Daybue (trofinetide) for the treatment of Rett syndrome in adults and children ages 2 and older who weigh at least 9 kg (about 19.8 lbs). The decision makes Daybue the first approved Rett treatment in Canada, according to its developer Acadia Pharmaceuticals.

Daybue (trofinetide) last year became the first treatment for Rett syndrome to win approval from the U.S. Food and Drug Administration (FDA), and it’s currently up for approval in Canada. Now, the company that sells it is working to get the therapy approved in other markets.

Treatment with ADH-503, an experimental oral treatment for pancreatic cancer, was found to slow disease progression and prolong survival in a mouse model of Rett syndrome, according to a study that focused on how brain immune cells may play a role in Rett. “I hope this work will ‘move…