Potential Rett syndrome drug shows promising results in phase 3 trial

This episode features a discussion of the need for meaningful therapies in Rett syndrome (RTT), and highlights the Lavender™ study, a phase 3, 12-week, double-blind, randomized, placebo-controlled study of the drug trofinetide in 187 young females, ages 5 to 20, with Rett syndrome. Investigators discuss public, top-level results of the potential first-of-its-kind drug to treat the symptoms of RTT, and share the hope that this trial paves the way for other successful trials in RTT and related disorders.

VKC Researchers: 

• Jeffrey L. Neul, M.D., Ph.D., Annette Schaffer Eskind Chair and Director, Vanderbilt Kennedy Center; Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education
• Cary Fu, M.D., Assistant Professor of Pediatrics, Pediatric Neurology