March 10 2023

FDA Approves Trofinetide for Treatment of Rett Syndrome!

Friday was a milestone day for our community, more than a decade in the making. Acadia Pharmaceuticals announced that Trofinetide, marketed under the new name DAYBUE™, was approved by the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. This is the first treatment ever approved for individuals with Rett, a drug that has demonstrated statistically significant improvement for participants in several key areas through multiple studies.

It's worth saying again - we would not be here without you! Whether you courageously participated in the clinical trial process or lent your support in other ways, we would not be celebrating today without the bravery of the families who selflessly gave their time to this process. The entire Rett community thanks you for making this day a reality.