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FDA approves Rett syndrome treatment 




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UNC Child Neurology Researchers Led UNC Trial Site for Rett ... - UNC Health and UNC School of Medicine

Yael Shiloh-Malawsky, MD, co-investigators Diana Cejas, MD, MPH, and Jane Fan, MD, and study coordinator Yulissa Gonzalez led the UNC School of Medicine site for a clinical trial of a FDA-approved Rett syndrome treatment from Acadia Pharmaceuticals. Yael Shiloh-Malawsky, MD This spring, Acadia Pharmaceuticals Inc. announced that FDA approved DAYBUE (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. DAYBUE is the first and only drug approved for the treatment of Rett syndrome. The UNC School of Medicine was part of this multi-site clinical trial, led by Yael Shiloh-Malawsky, MD, associate professor of neurology.

news.unchealthcare.org     Apr 27, 2023