Multisite clinical trial will compare three FDA-approved drugs 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.
Affecting 1 in 10,000 females at birth, and males even more rarely, Rett syndrome is a rare genetic neurodevelopmental disorder that affects brain development.
“It robs affected individuals of the ability to use their hands or speak and causes problems with mobility, as well as a number of other issues,” said Jeffrey Neul, Annette Schaffer Eskind Professor, ...














