Rett syndrome

January 9, 2024

VUMC receives $13 million grant to coordinate multisite clinical trial for Rett syndrome treatment

Vanderbilt University Medical Center received a $13 million Department of Defense grant to lead a multisite clinical trial that will evaluate repurposed FDA-approved drugs as treatment options for patients with Rett syndrome.

By Danny Bonvissuto
July 7, 2023

Rett syndrome drug studied at Vanderbilt approved for patients

The first drug to treat the symptoms of Rett syndrome was recently approved, following a Nature Medicine study publishing results from the pivotal phase 3 LAVENDER study led by investigators from Vanderbilt University Medical Center to evaluate efficacy and safety of trofinetide.

By Craig Boerner
December 6, 2021

Potential Rett syndrome drug shows promising results in phase 3 trial

A potential first-of-its-kind drug to treat the symptoms of Rett syndrome received positive top-line results from a phase 3 trial led by investigators from Vanderbilt University Medical Center to evaluate efficacy and safety.

By Craig Boerner
June 6, 2019

Treating core Rett syndrome symptoms

A new study published in Neurology reports the drug trofinetide has proven safe and effective in treating core symptoms of Rett syndrome in female children and adolescents.

By Kelsey Herbers
Caucasian mom and toddler daughter playing in the park
August 24, 2017

Drug discovery efforts may lead to new Rett syndrome treatments

Vanderbilt University research-ers have relieved symptoms of Rett syndrome in a mouse model with a small molecule that works like the dimmer switch in an electrical circuit.

By Bill Snyder
December 13, 2016

Rett syndrome expert Neul to speak at Kennedy Center on Friday

Jeffrey Neul, M.D., Ph.D., division head of Child Neurology and vice chair for Developmental Neurosciences at the University of California, San Diego, will present a lecture on Friday, Dec. 16, at Vanderbilt Kennedy Center (VKC).

By VUMC News and Communications
November 17, 2016

VUMC’s Rett Syndrome Clinic lands national recognition

Vanderbilt’s Rett Syndrome Clinic has been named a Rett Syndrome Clinical Research Center of Excellence by Rettsyndrome.org.
By VUMC News and Communications
April 14, 2016

Zoghbi to explore Rett Syndrome at upcoming Vanderbilt Prize Lecture

Huda Zoghbi, M.D., recipient of the 2015 Vanderbilt Prize in Biomedical Science, will deliver her Vanderbilt Prize Lecture on Thursday, April 21.
By Bill Snyder
March 3, 2016

Study reveals possible ‘dimmer switch’ drug for Rett syndrome

Researchers at Vanderbilt University Medical Center have relieved symptoms in a mouse model of Rett syndrome with a drug-like compound that works like the dimmer switch in an electrical circuit.
By Bill Snyder
January 23, 2014

Autism Speaks grant boosts Rett syndrome research

Colleen Niswender, Ph.D., research associate professor of Pharmacology, has received a three-year, $450,000 grant from the autism science and advocacy organization Autism Speaks to support studies investigating a possible new treatment for Rett syndrome.
By Bill Snyder