Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2011 Aug 17;2(8):402-10.
doi: 10.1021/cn200019z. Epub 2011 Mar 22.

Fragile X syndrome: an update on developing treatment modalities

Aileen Healy  1 , Roger Rush, Timothy Ocain
Affiliations
Review

Fragile X syndrome: an update on developing treatment modalities

Aileen Healy et al. ACS Chem Neurosci. .

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.

PubMed Disclaimer

Figures

Figure 1
Figure 1
Structures of prototypical mGluR5 NAMs 1−3, and novel chemotypes 4−14 indicating the company/university that developed them.
See this image and copyright information in PMC

References

    1. Martin J.; Bell J. (1943) A pedigree of mental defect showing sex-linkage. J. Neurol. Neurosurg. Psychiatry 6, 154–157. - PMC - PubMed
    1. Sutherland G. R. (1977) Fragile sites on human chromosomes: demonstration of their dependence on the type of tissue culture medium. Science 197, 265–266. - PubMed
    1. Verkerk A. J.; Pieretti M.; Sutcliffe J. S.; Fu Y. H.; Kuhl D. P.; Pizzuti A.; Reiner O.; Richards S.; Victoria M. F.; Zhang F. P. (1991) Identification of a gene (FMR-1) containing a CGG repeat coincident with a breakpoint cluster region exhibiting length variation in fragile X syndrome. Cell 65, 905–914. - PubMed
    1. Crawford D. C.; Acuna J. M.; Sherman S. L. (2001) FMR1 and the fragile X syndrome: human genome epidemiology review. Genet. Med. 3, 359–371. - PMC - PubMed
    1. Symons F. J.; Byiers B. J.; Raspa M.; Bishop E.; Bailey D. B. (2010) Self-injurious behavior and fragile X syndrome: findings from the national fragile X survey. Am. J. Intellect. Dev. Disability 115, 473–481. - PubMed

MeSH terms

Substances