Special Episode: Trofinetide Approved for Rett Syndrome

Jeffrey L. Neul, MD, PhD

A special episode of the NeurologyLive^® Mind Moments^® podcast is now live! Scroll down to listen or click here to subscribe on your favorite streaming service.

The Mind Moments^® podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis (MS), Parkinson disease, dementia, sleep disorders, and more.

This episode, "Trofinetide Approved for Rett Syndrome," features an exclusive interview with Jeffrey L. Neul, MD, PhD, the Annette Schaffer Eskind Chair and director of the Vanderbilt Kennedy Center, and professor of pediatrics at Vanderbilt University Medical Center, and the lead investigator on the phase 3 study of the drug, called LAVENDER (NCT04181723). Trofinetide, now branded as Daybue by Acadia Pharmaceuticals, is the first treatment approval for Rett syndrome, marking a milestone in the field of developmental disorder care.

Click here to read more of NeurologyLive^®'s coverage of the trofinetide approval.

EPISODE BREAKDOWN

• 0:30 – Trofinetide (Daybue; Acadia) approved for Rett syndrome
• 1:30 – Jeffrey L. Neul, MD, PhD, on the approval
• 4:10 – Prevalence of Rett syndrome and the impact on research
• 5:40 – Supporting data from the phase 3 LAVENDER study
• 7:20 – Safety data from the clinical development and managment of common adverse events
• 8:45 – Closing thoughts

Click here to subscribe to the Mind Moments^® podcast. Be sure to leave a rating and review for the show. Thanks for listening!

REFERENCES
1. Acadia Pharmaceuticals Announces U.S. FDA Approval of DAYBUE™ (trofinetide) for the Treatment of Rett Syndrome in Adult and Pediatric Patients Two Years of Age and Older News Release. Acadia Pharmaceuticals. March 10, 2023. Accessed March 13, 2023. https://acadia.com/media/news-releases/acadia-pharmaceuticals-announces-u-s-fda-approval-of-daybue-trofinetide-for-the-treatment-of-rett-syndrome-in-adult-and-pediatric-patients-two-years-of-age-and-older/
2. Neul JL. Rett Syndrome. National Organization for Rare Diseases. Updated December 17, 2019. Accessed March 13, 2023. https://rarediseases.org/rare-diseases/rett-syndrome/