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    Novel end points for clinical trials in young children with cystic fibrosis

    Access Status
    Fulltext not available
    Authors
    Simpson, S.
    Mott, L.
    Esther, C.
    Stick, S.
    Hall, Graham
    Date
    2013
    Type
    Journal Article
    
    Metadata
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    Citation
    Simpson, S. and Mott, L. and Esther, C. and Stick, S. and Hall, G. 2013. Novel end points for clinical trials in young children with cystic fibrosis. Expert Review of Respiratory Medicine. 7 (3): pp. 231-243.
    Source Title
    Expert Review of Respiratory Medicine
    DOI
    10.1586/ers.13.25
    ISSN
    1747-6348
    School
    School of Physiotherapy and Exercise Science
    URI
    http://hdl.handle.net/20.500.11937/54974
    Collection
    • Curtin Research Publications
    Abstract

    Cystic fibrosis (CF) lung disease commences early in the disease progression and is the most common cause of mortality. While new CF disease-modifying agents are currently undergoing clinical trial evaluation, the implementation of such trials in young children is limited by the lack of age-appropriate clinical trial end points. Advances in infant and preschool lung function testing, imaging of the chest and the development of biochemical biomarkers have led to increased possibility of quantifying mild lung disease in young children with CF and objectively monitoring disease progression over the course of an intervention. Despite this, further standardization and development of these techniques is required to provide robust objective measures for clinical trials in this age group.

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