Rett Syndrome Tomorrow: Management Options on the Horizon

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, PhDInstructorDepartment of NeurologyHarvard Medical SchoolAttending PhysicianChild NeurologyBoston Children’s HospitalBoston, Massachusetts

Jeffrey Neul, MD, PhDDirectorPediatricsVanderbilt University Medical CenterNashville, Tennessee

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

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