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Clarifying the "Vanderbilt Breakthrough Story"

April 29, 2016

On April 26th, WBRC Fox 6 News covered a story with the headline "Vanderbilt researchers announce breakthroughs treating Rett syndrome”. RSRT received an influx of emails and phone calls asking for more information. So we decided to put this short post up to efficiently answer your questions.

Most people reading the story and watching the video may surmise that the compound(s) being discussed had quickly progressed from mouse studies to phase 2 clinical trials in people and that beautiful two-year-old Savannah was in the trial and regaining lost skills.


1. The compounds being developed for Rett Syndrome in the lab of Colleen Niswender have to date been tested only in mice, not people. The journalist erroneously jumps from mouse to people and says “….Niswender says those compounds are showing remarkable improvements in Rett patients”.

2. The Phase 2 trial mentioned in the story and video refers to the Neuren drug, Trofinetide, not the compounds being developed at Vanderbilt.

3. The two compounds that Niswender hopes will be in clinical trials by the end of the year are for other disorders, not Rett Syndrome. The compounds they are developing for Rett are in an earlier development phase.

4. Beautiful Savannah is improving due to therapies, not drug treatment. The article states: Thanks to regular physical, occupational, speech and feeding therapies, Savannah is also improving.

What is accurate is that the research group at Vanderbilt is top-notch and they are indeed interested in Rett Syndrome and developing compounds that may target some of the symptoms. If and when that happens that will certainly be a breakthrough to celebrate.