Multisite Clinical Trial Will Evaluate Three FDA-Approved Drugs As Treatment Options for Rett Syndrome

According to a January 16, 2023 news release, Vanderbilt University Medical Center (VUMC) has 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, MD, PhD, Annette Schaffer Eskind Professor, Director of the Vanderbilt Kennedy Center and the grant’s principal investigator. “People with Rett syndrome require lifelong care for activities of daily living. Although symptom management, therapies, and a newly FDA-approved drug can help, there remains a great need to find new therapies to improve the lives of people with Rett syndrome.” 

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