“The Promise of Discovery” is a podcast hosted by the Vanderbilt Kennedy Center (VKC) that highlights research in intellectual and developmental disabilities. The goals of the podcast are to host conversations about research in plain language and to highlight “real world” implications. The discussions cover why the research matters in the lives of people with disabilities and families, how the research impacts and enhances what we know already and/or raises additional questions in the field, and what implications the research might have for policy.
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“The Promise of Discovery” is a podcast hosted by the Vanderbilt Kennedy Center (VKC) that highlights research in intellectual and developmental disabilities. The goals of the podcast are to host conversations about research in plain language and to highlight “real world” implications. The discussions cover why the research matters in the lives of people with disabilities and families, how the research impacts and enhances what we know already and/or raises additional questions in the field, and what implications the research might have for policy.
Potential Rett syndrome drug shows promising results in phase 3 trial
The Promise of Discovery
25 minutes
3 years ago
Potential Rett syndrome drug shows promising results in phase 3 trial
The Promise of Discovery Season 2, Episode 7:
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
The Promise of Discovery
“The Promise of Discovery” is a podcast hosted by the Vanderbilt Kennedy Center (VKC) that highlights research in intellectual and developmental disabilities. The goals of the podcast are to host conversations about research in plain language and to highlight “real world” implications. The discussions cover why the research matters in the lives of people with disabilities and families, how the research impacts and enhances what we know already and/or raises additional questions in the field, and what implications the research might have for policy.
