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The study by Glaze et al.1 in this issue of Neurology® provides a remarkable glimpse into the future of targeted treatments for neurodevelopmental disorders. The investigators are to be commended for their well-designed clinical trial of trofinetide, an analog of the amino terminal tripeptide of the insulin-like growth factor 1, in the treatment of girls with Rett syndrome, a rare severe disabling disorder for which there is no current treatment. Their study provides Class I evidence that trofinetide is well-tolerated and effective at ameliorating specific core symptoms of Rett syndrome. This article is a positive light in a sea of negative studies for other neurodevelopmental disorders.2–5
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Go to Neurology.org/N for full disclosures. Funding information and disclosures deemed relevant by the authors, if any, are provided at the end of the editorial.
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