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Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

bioRxiv

Dec 8, 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
Study
Clinical Trial [Publication Type]
Clinical Trials
Corticosteroid nasal preparations for topical use
Mutation Abnormality
Vitamin D
Ambulation
Pharmaceutical Preparations
Corticosteroid otologicals
3
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  • Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

    bioRxiv

    Dec 8, 2014

    Richard T WangStanley F. Nelson

    PMID: 990012344

    DOI: 10.1101/012344

    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
    Study
    Clinical Trial [Publication Type]
    Clinical Trials
    Corticosteroid nasal preparations for topical use
    3

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