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

2014-12-08

Abstract excerpt

<h4>ABSTRACT</h4> 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. Data collected were compared to prior natural history data in regard to age at diagnosis, mutation spectrum, and...

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Literature Corpus work
d10c11f2-b4f7-5814-a3ca-c5457dc35a12
DOI
10.1101/012344
Open publication

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