Marisa Wexler, MS,  senior science writer—

The Embolden clinical trial, a registrational study testing Neurogene‘s gene therapy NGN-401 in people with Rett syndrome, has begun dosing participants. “Dosing the first participant in the Embolden registrational trial of NGN-401 marks a major milestone in advancing a gene therapy for Rett syndrome,” Rachel McMinn, PhD, Neurogene’s…

After expatriation of a partnership agreement with Astellas, Taysha Gene Therapies regained full rights to TSHA-102, its experimental gene therapy being developed to treat Rett syndrome. “We are thrilled to regain full global rights to our lead TSHA-102 Rett syndrome program, which enables full strategic flexibility and optionality…

A gene therapy showing potential to restore lost developmental milestones in people living with Rett syndrome has earned breakthrough therapy status from the U.S. Food and Drug Administration (FDA). The experimental therapy, TSHA-102, is being developed by Taysha Gene Therapies. A breakthrough therapy designation…

October is Rett Syndrome Awareness Month, and supporters are going purple to bring attention to the rare genetic condition. “There’s no one right way to raise awareness,” the International Rett Syndrome Foundation (IRSF) says on its website. “Whether you’re posting on social media, sharing your story, or just starting…

Many types of movement disorders such as uncontrolled muscle contractions and Parkinson’s-like motor changes often co-occur with Rett syndrome, a study shows. The severity of movement disorders is associated with Rett’s overall severity, with uncontrolled muscle contractions particularly linked with breathing trouble and digestive problems, data indicate. The findings…

Unravel Biosciences is asking authorities in Colombia to allow the launch — which could be as early as this summer, if cleared — of proof-of-concept clinical trials in the country to test RVL001, its treatment candidate for Rett syndrome. The company has submitted an application seeking regulatory clearance…

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…