Marisa Wexler, MS,  senior science writer—

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…

Long-term treatment with Daybue (trofinetide) led to continual improvements among children and young adults with Rett syndrome in extension studies, with patients experiencing notable benefits in communication and other relevant issues, according to new data. Slightly more than a year ago, Daybue became the first treatment for Rett…

When clinicians have to decide whether to recommend invasive ventilation for adolescents with Rett syndrome, parental and familial support are the biggest factors in their decision, a recent study reports. Many teenagers with Rett syndrome have difficulty breathing, and clinicians for some may recommend invasive ventilation via a tracheostomy…

The U.S. Food and Drug Administration (FDA) has given a regenerative medicine advanced therapy (RMAT) designation to TSHA-102, an experimental gene therapy for Rett syndrome. RMAT is given to regenerative medicines — cell therapies, therapeutic tissues, human cell and tissue products, or any combination using such therapies or products…