News

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…

Ferroptosis, a form of cell death marked by the accumulation of iron, may contribute to cellular dysfunction in Rett syndrome, according to a study. Cells from Rett patients exhibited hallmarks of ferroptosis, including oxidative damage to lipids, or fats, and abnormal mitochondria, the cell’s powerhouses. Incubation with a…

Researchers in Italy described a rare case of 27-year-old identical twin sisters with Rett syndrome who, despite sharing the same disease-causing mutation, developed symptoms of different severity. Identical (monozygotic) twins develop from a single fertilized egg that divides into two embryos, so they are genetically identical. “The clinical differences…

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…

Neurogene expects dosing for a registrational clinical trial of its investigational gene therapy NGN-401 for people with Rett syndrome will start by the end 2025. The U.S. Food and Drug Administration (FDA) agreed to key elements of the trial design over the summer, and Neurogene reports it…

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…

Mutations in the MECP2 gene — which typically underlie Rett syndrome — cause problems in how neurons, or nerve cells, produce energy and communicate, and affect how well they can repair breaks to DNA. That’s according to the findings of a new study, done in both human brain cells…

One year of daily Daybue (trofinetide) treatment led to improvements in behaviors such as nonverbal communication, alertness, and social interaction and connectedness among children and adults with Rett syndrome. That’s according to published interim data from Acadia Pharmaceuticals‘ Phase 4 LOTUS study that assessed the safety and…

Using an oral cannabidiol (CBD) solution as an add-on treatment to antiseizure medications was shown to lower the frequency of drug-resistant seizures by more than half in about two-thirds of children with Rett syndrome in a study conducted in Italy. “Our findings suggest a strong trend toward the efficacy…