News

Early changes in speech, including fewer varied sounds, may signal Rett syndrome before obvious developmental delays and this may help doctors reach a diagnosis sooner, a study of twin girls who didn’t share the same genetic makeup suggests. One twin developed normally, but the other, who was later…

A new study highlights how MeCP2 protein dysfunction, particularly related to changes in alternative splicing — a process by which different proteins can be made from the same gene — implicated in brain function, may contribute to Rett syndrome. Specifically, the researchers found that in both Rett patients and…

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,…

The protocol of the Phase 1/2 trial (NCT05898620) that’s evaluating Neurogene’s NGN-401 in girls with Rett syndrome has been amended to use the low treatment dose of the gene therapy for all future participants, the company announced. The protocol alteration was submitted to the U.S.  Food and Drug…

One and a half years of daily Daybue (trofinetide) treatment safely improved communication, eye contact, and hand use among girls ages 2-4 with Rett syndrome, according to the final published results of the DAFFODIL trial, which tested the approved therapy in the youngest of patients. Although most patients…

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…