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

Andrew Ligsay; Randi Hagerman; Elizabeth Berry-Kravis

doi:10.1016/B978-0-12-804461-2.00019-6

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

Andrew Ligsay^*, Randi Hagerman, Elizabeth Berry-Kravis

^*Corresponding author for this work

Pediatrics

Research output: Chapter in Book/Report/Conference proceeding › Chapter

4 Scopus citations

Abstract

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 language	English (US)
Title of host publication	Fragile X Syndrome
Subtitle of host publication	From Genetics to Targeted Treatment
Publisher	Elsevier
Pages	401-418
Number of pages	18
ISBN (Electronic)	9780128045077
ISBN (Print)	9780128044612
DOIs	https://doi.org/10.1016/B978-0-12-804461-2.00019-6
State	Published - May 26 2017

Keywords

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

ASJC Scopus subject areas

General Neuroscience

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10.1016/B978-0-12-804461-2.00019-6

Cite this

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abstract = "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.",

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

Abstract

Keywords

ASJC Scopus subject areas

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