Document Detail


Advances in the treatment of fragile X syndrome.
MedLine Citation:
PMID:  19117905     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
The FMR1 mutations can cause a variety of disabilities, including cognitive deficits, attention-deficit/hyperactivity disorder, autism, and other socioemotional problems, in individuals with the full mutation form (fragile X syndrome) and distinct difficulties, including primary ovarian insufficiency, neuropathy and the fragile X-associated tremor/ataxia syndrome, in some older premutation carriers. Therefore, multigenerational family involvement is commonly encountered when a proband is identified with a FMR1 mutation. Studies of metabotropic glutamate receptor 5 pathway antagonists in animal models of fragile X syndrome have demonstrated benefits in reducing seizures, improving behavior, and enhancing cognition. Trials of metabotropic glutamate receptor 5 antagonists are beginning with individuals with fragile X syndrome. Targeted treatments, medical and behavioral interventions, genetic counseling, and family supports are reviewed here.
Authors:
Randi J Hagerman; Elizabeth Berry-Kravis; Walter E Kaufmann; Michele Y Ono; Nicole Tartaglia; Ave Lachiewicz; Rebecca Kronk; Carol Delahunty; David Hessl; Jeannie Visootsak; Jonathan Picker; Louise Gane; Michael Tranfaglia
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Review    
Journal Detail:
Title:  Pediatrics     Volume:  123     ISSN:  1098-4275     ISO Abbreviation:  Pediatrics     Publication Date:  2009 Jan 
Date Detail:
Created Date:  2009-01-01     Completed Date:  2009-02-06     Revised Date:  2010-12-03    
Medline Journal Info:
Nlm Unique ID:  0376422     Medline TA:  Pediatrics     Country:  United States    
Other Details:
Languages:  eng     Pagination:  378-90     Citation Subset:  AIM; IM    
Affiliation:
MIND. Institute, University of California Davis, School of Medicine, Sacramento, CA 95817, USA. randi.hagerman@ucdmc.ucdavis.edu
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MeSH Terms
Descriptor/Qualifier:
Animals
Excitatory Amino Acid Antagonists / therapeutic use
Fragile X Syndrome / diagnosis*,  genetics,  therapy*
Gene Therapy / trends
Humans
Mutation / genetics
Grant Support
ID/Acronym/Agency:
1KL2RR025009/RR/NCRR NIH HHS; 5K12RR017643/RR/NCRR NIH HHS; AG032115/AG/NIA NIH HHS; DE019583/DE/NIDCR NIH HHS; HD02274/HD/NICHD NIH HHS; HD036071/HD/NICHD NIH HHS; HD24061/HD/NICHD NIH HHS; MH77554/MH/NIMH NIH HHS; R01 HD036071-11/HD/NICHD NIH HHS; RL1 AG032115-02/AG/NIA NIH HHS; RR024146/RR/NCRR NIH HHS
Chemical
Reg. No./Substance:
0/Excitatory Amino Acid Antagonists
Comments/Corrections

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