Turning the tide on targeted treatments for neurodevelopmental disorders

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

from Neurology recent issues http://bit.ly/2IlEBzW