Note: This story was updated Dec. 5, 2025, to correct the primary outcome in the Embolden study. NGN-401, an experimental…
Articles by Marisa Wexler, MS
The Embolden clinical trial, a registrational study testing Neurogene‘s gene therapy NGN-401 in people with Rett syndrome, has…
After expatriation of a partnership agreement with Astellas, Taysha Gene Therapies regained full rights to TSHA-102, its experimental gene…
A gene therapy showing potential to restore lost developmental milestones in people living with Rett syndrome has earned breakthrough…
October is Rett Syndrome Awareness Month, and supporters are going purple to bring attention to the rare genetic condition. “There’s…
Many types of movement disorders such as uncontrolled muscle contractions and Parkinson’s-like motor changes often co-occur with Rett syndrome,…
Unravel Biosciences is asking authorities in Colombia to allow the launch — which could be as early as this…
NGN-401, a gene therapy candidate for Rett syndrome that’s now being tested in a Phase 1/2 clinical trial, was…
Treatment with NA-921 was found to improve measures of abnormal behavior and overall health in girls and young women with…
Dysfunction of an RNA molecule called NEAT1 contributes to nerve cell damage in Rett syndrome, a study suggested, implying…