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    New Therapeutic Options for Fragile X Syndrome

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    Authors
    Jalnapurkar, Isha
    Cochran, David M.
    Frazier, Jean A.
    UMass Chan Affiliations
    Department of Psychiatry
    Eunice Kennedy Shriver Center
    Document Type
    Journal Article
    Publication Date
    2019-02-27
    Keywords
    Drug development
    Fragile X syndrome
    Pharmacotherapy
    Supportive therapies
    Targeted treatments
    Congenital, Hereditary, and Neonatal Diseases and Abnormalities
    Mental and Social Health
    Nervous System Diseases
    Neurology
    Pediatrics
    Pharmaceutics and Drug Design
    Psychiatry
    Psychiatry and Psychology
    Therapeutics
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    Link to Full Text
    https://doi.org/10.1007/s11940-019-0551-8
    Abstract
    PURPOSE OF REVIEW: The purpose of this review is to provide an overview of current research and clinical practice guidelines in fragile X syndrome (FXS) with regard to therapeutic approaches in the management of this condition. The authors summarize and discuss findings from relevant preclinical studies and results from clinical trials in human subjects with FXS. Additionally, we provide an outline of the basic framework for understanding and providing educational and psychosocial supports for these individuals. RECENT FINDINGS: Current treatments in FXS are largely symptom based and focused on managing associated psychiatric and behavioral co-morbidities. While data from animal studies has been promising in providing targeted treatments to correct the underlying deficits at the cellular level, there have not been as robust findings in human trials. There are several targeted treatments for FXS currently under development. SUMMARY: Individuals with FXS present with several behavioral challenges including anxiety, social withdrawal, ADHD, hyperarousal, self-injury, and aggression. Therapeutic services are often necessary, such as behavioral intervention, speech and language therapy, occupational therapy, and individualized educational support; adjunctive psychopharmacologic treatment is often helpful as well. It is important to address these symptoms and weigh the evidence for the use of medications that target the underlying neurobiology and pathophysiology of the syndrome.
    Source

    Curr Treat Options Neurol. 2019 Feb 27;21(3):12. doi: 10.1007/s11940-019-0551-8. Link to article on publisher's site

    DOI
    10.1007/s11940-019-0551-8
    Permanent Link to this Item
    http://hdl.handle.net/20.500.14038/46309
    PubMed ID
    30809735
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    Link to Article in PubMed

    ae974a485f413a2113503eed53cd6c53
    10.1007/s11940-019-0551-8
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