Fragile X syndrome: an update on developing treatment modalities

ACS Chem Neurosci. 2011 Aug 17;2(8):402-10. doi: 10.1021/cn200019z. Epub 2011 Mar 22.

Abstract

Intellectual disability (ID; mental retardation) is considered an immutable condition. Current medical practices are aimed at relieving symptoms and not at altering the underlying cognitive deficits. Scientific advancements from the past decade have led to the exciting possibility that ID may now be treatable. Moreover, pharmaceutical therapies targeting the most common form of inherited ID, Fragile X syndrome (FXS), may become the new benchmark for central nervous system (CNS) drug discovery: seeking cures for neurodevelopmental disorders.

Keywords: Fragile X syndrome; fragile X mental retardation gene; fragile X mental retardation protein; metabotropic glutamate receptor 5; γ-aminobutyric acid.

Publication types

  • Review

MeSH terms

  • Allosteric Regulation / drug effects
  • Allosteric Regulation / physiology
  • Animals
  • Baclofen / analogs & derivatives
  • Baclofen / chemistry
  • Baclofen / pharmacology
  • Baclofen / therapeutic use
  • Central Nervous System Agents / chemistry*
  • Central Nervous System Agents / pharmacology
  • Central Nervous System Agents / therapeutic use*
  • Drug Discovery / trends*
  • Fragile X Syndrome / drug therapy*
  • Fragile X Syndrome / metabolism
  • Fragile X Syndrome / psychology
  • Humans
  • Receptor, Metabotropic Glutamate 5
  • Receptors, Metabotropic Glutamate / agonists
  • Receptors, Metabotropic Glutamate / antagonists & inhibitors
  • Receptors, Metabotropic Glutamate / physiology*
  • Treatment Outcome

Substances

  • Central Nervous System Agents
  • Receptor, Metabotropic Glutamate 5
  • Receptors, Metabotropic Glutamate
  • Baclofen
  • arbaclofen placarbil