News for Healthier Living

Study Aims to Establish Key Biomarkers in Rett Syndrome

Like many neurodevelopmental disorders, Rett syndrome has long had a lack of disease-specific treatments. But that landscape is poised to change. The Food and Drug Administration approved the first treatment for Rett, trofinetide, in 2023, and other novel therapies--including a potential gene therapy--are in the pipeline.

May 15, 2025


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