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Online self-report data for duchenne muscular dystrophy confirms natural history and can be used to assess for therapeutic benefits

PLoS Currents

Oct 17, 2014

Richard T WangStanley F. Nelson

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Abstract

To assess the utility of online patient self-report outcomes in a rare disease, we attempted to observe the effects of corticosteroids in delaying age at fulltime wheelchair use in Duchenne muscular dystrophy (DMD) using data from 1,057 males from DuchenneConnect, an online registry. Da...read more

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Corticosteroid ophthalmologic and otologic preparations
Clinical Trial [Publication Type]
Corticosteroid nasal preparations for topical use
Mutation Abnormality
Vitamin D
Ambulation
Gait Disorders, Neurologic
Corticosteroid otologicals
Muscular Dystrophy, Duchenne
Prednisone
1
1
26
Paper Details
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  • Online self-report data for duchenne muscular dystrophy confirms natural history and can be used to assess for therapeutic benefits

    PLoS Currents

    Oct 17, 2014

    Richard T WangStanley F. Nelson

    PMID: 25635234

    DOI: 10.1371/currents.md.e1e8f2be7c949f9ffe81ec6fca1cce6a

    Abstract

    To assess the utility of online patient self-report outcomes in a rare disease, we attempted to observe the effects of corticosteroids in delaying age at fulltime wheelchair use in Duchenne muscular dystrophy (DMD) using data from 1,057 males from DuchenneConnect, an online registry. Da...read more

    Mentioned in this Paper

    Corticosteroid ophthalmologic and otologic preparations
    Clinical Trial [Publication Type]
    Corticosteroid nasal preparations for topical use
    Mutation Abnormality
    Vitamin D
    1
    1
    26
    Paper Details
    References
    • References
    • Citations10

      References currently unavailable

      We're still populating references for this paper, please check back later.
    • References
    • Citations10
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