Future Options for RTT

CE / CME

Rett Syndrome Tomorrow: Management Options on the Horizon

Pharmacists: 0.50 contact hour (0.05 CEUs)

Physician Assistants/Physician Associates: 0.50 AAPA Category 1 CME credit

Nurses: 0.50 Nursing contact hour, including 0.50 hour of pharmacotherapy credit 

Physicians: Maximum of 0.50 AMA PRA Category 1 Credit

Released: March 27, 2023

Expiration: March 26, 2024

David N Lieberman
David N Lieberman, MD, PhD
Jeffrey Neul
Jeffrey Neul, MD, PhD

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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, a newly approved treatment option for Rett syndrome, and blarcamesine, an agent in late-stage clinical trials 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.