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Treating core Rett syndrome symptoms | VUMC Reporter

by Kelsey Herbers

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.

A neurodevelopmental disorder, Rett syndrome is characterized by normal early growth followed by a loss of use of the hands, loss of spoken language, problems with walking and repetitive hand movements. There are currently no treatments available to relieve prevalent symptoms.

In the double-blind, placebo-controlled study, Jeffrey Neul, MD, PhD, and colleagues studied the tolerability, pharmacokinetics and clinical response of trofinetide in children ages 5-15 who received varying doses. All doses were well-tolerated, with children who received the highest dose showing significant improvements in behavior and other clinical features relative to those randomized to the placebo.

The study indicates trofinetide’s potential for treating patients with Rett syndrome, supporting additional trials of this medication in patients with the disorder.

This research was supported by Neuren Pharmaceuticals and Rettsyndrome.org.


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