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April 16, 2019; 92 (16) Editorial

Turning the tide on targeted treatments for neurodevelopmental disorders

Randi Hagerman, Roberto Tuchman
First published March 27, 2019, DOI: https://doi.org/10.1212/WNL.0000000000007301
Randi Hagerman
From the MIND Institute and Department of Pediatrics (R.H.), University of California Davis Medical Center, Sacramento; and Department of Neurology (R.T.), Nicklaus Children's Hospital, Miami, FL.
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Roberto Tuchman
From the MIND Institute and Department of Pediatrics (R.H.), University of California Davis Medical Center, Sacramento; and Department of Neurology (R.T.), Nicklaus Children's Hospital, Miami, FL.
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Turning the tide on targeted treatments for neurodevelopmental disorders
Randi Hagerman, Roberto Tuchman
Neurology Apr 2019, 92 (16) 741-742; DOI: 10.1212/WNL.0000000000007301

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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.

  • See page 754

  • © 2019 American Academy of Neurology
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