Rett gene therapy NGN-401 chosen for FDA's START pilot program
NGN-401 is one of six picked, three each by the Center for Biologics Evaluation and Research, and the Center for Drug Evaluation and Research.| Rett Syndrome News
Our Gene Therapy Platform Technology (EXACT™) delivers highly controlled, consistent levels of transgene expression.| Neurogene
NGN-401 is one of six picked, three each by the Center for Biologics Evaluation and Research, and the Center for Drug Evaluation and Research.| Rett Syndrome News
Study record managers: refer to the Data Element Definitions if submitting registration or results information.| clinicaltrials.gov
Marisa Wexler is a senior science writer for Rett Syndrome News with an MS in cellular and molecular pathology. She covers the latest news and information on a variety of Rett syndrome topics.| Rett Syndrome News
Learn more about what causes Rett syndrome, a rare genetic disorder that mostly affects females.| Rett Syndrome News
Category archive page for News.| Rett Syndrome News
Long-term treatment with Daybue (trofinetide) led to continual improvements in symptoms in people with Rett syndrome in extension studies, data show.| Rett Syndrome News
Learn more about the rare neurodevelopmental disorder Rett syndrome, including its causes, symptoms, diagnosis and treatment.| Rett Syndrome News
