Rett syndrome is a rare but severe neurodevelopmental disorder, and current treatment approaches primarily focus on symptom management and supportive care. However, recent clinical developments are providing hope for a changing treatment landscape. In this podcast episode, experts in Rett syndrome David N. Lieberman, MD, PhD, and Jeffrey Neul, MD, PhD, discuss trofinetide and blarcamesine, 2 agents in late-stage clinical trials as treatment options for Rett syndrome. Throughout their conversation, Drs Lieberman and Neul review the pivotal LAVENDER, DAFFODIL, AVATAR, and EXCELLENCE trials, including information on the safety and efficacy profiles of trofinetide and blarcamesine. The experts end their discussion with a conversation about the potential roles of ketamine and gene therapy in the management of Rett syndrome.      

Presenters:

David N. Lieberman, MD, PhD
Instructor
Department of Neurology
Harvard Medical School
Attending Physician
Child Neurology
Boston Children’s Hospital
Boston, Massachusetts

Jeffrey Neul, MD, PhD
Director
Pediatrics
Vanderbilt University Medical Center
Nashville, Tennessee

This activity is supported by educational funding provided by Acadia Pharmaceuticals Inc.

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