CCO Neuroscience Podcast

What’s New in the Treatment of Rett Syndrome?

Episode Summary

Listen to this podcast episode for a 2024 update on approved and investigational treatment for patients with Rett syndrome from 2 experts in pediatric neurology.

Episode Notes

In this episode, David N. Lieberman, MD, PhD, and Eric Marsh, MD, PhD, discuss recent updates relevant to the treatment of Rett syndrome, including:

• The recently updated Comprehensive Care Guidelines from the International Rett Syndrome Foundation, available at rettsyndrome.org
• Ongoing collection and analysis of longitudinal data within the Rett Syndrome Natural History Study
• Efficacy and safety data from the double-blind, placebo-controlled phase III registrational LAVENDER trial evaluating oral trofinetide, a small molecule analogue of insulin-like growth factor 1
• Topline results from phase III AVATAR and EXCELLENCE trials of the sigma-1 receptor agonist blarcamesine
• Several early ongoing gene therapy trials

Dr Lieberman and Dr Marsh complement this informative update with wisdom from their own clinical experience to provide a full picture of current treatment and management options available to patients with Rett syndrome.

Presenters: 

David N. Lieberman, MD, PhD
Director, Comprehensive Rett Syndrome Clinic
Department of Neurology
Boston Children's Hospital
Boston, Massachusetts

Eric Marsh, MD PhD
Clinical Director, Orphan Disease Center
Director (MPI), CHOP/Penn IDDRC
Professor, Departments of Neurology and Pediatrics
Division of Neurology
Children’s Hospital of Philadelphia
Perelman School of Medicine
University of Pennsylvania
Philadelphia, Pennsylvania

This content is part of an online CME program in partnership with the International Rett Syndrome Foundation and supported by an educational grant from Acadia Pharmaceuticals. Link to full program:
https://bit.ly/4fJRRL0