Overview of Targeted Double-Blind, Placebo-Controlled Clinical Trials in Fragile X Syndrome

Andrew Ligsay, Randi J Hagerman, Elizabeth Berry-Kravis

Research output: Chapter in Book/Report/Conference proceedingChapter

4 Scopus citations


This chapter is an up-to-date review of targeted treatment trials for patients with fragile X syndrome (FXS). Specifically, there is a focus on controlled trials directed toward cellular receptors and drugs, such as AMPA receptor activators, mGluR5 negative allosteric modulators, and GABA receptor agonists. While only some patients show improvement during these studies, advances in FXS animal models are helping to uncover specific biomarkers that may help to identify patients who will be the most responsive. Finally, this chapter discusses the need for future controlled trials, regarding quantitative outcome measures and biomarkers, targeted of treatment for FXS.

Original languageEnglish (US)
Title of host publicationFragile X Syndrome
Subtitle of host publicationFrom Genetics to Targeted Treatment
Number of pages18
ISBN (Electronic)9780128045077
ISBN (Print)9780128044612
StatePublished - May 26 2017


  • Ampakines
  • Fragile X syndrome
  • GABA antagonists
  • Lithium
  • MGluR-NAMs
  • Minocycline
  • Sertraline
  • Targeted treatments
  • Trofinetide

ASJC Scopus subject areas

  • Neuroscience(all)


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