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

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. Data collected were compared to prior natural history data in regard to age at diagnosis, mutation spectrum, and age at loss of ambulation. Because registrants reported differences in steroid and other medication usage, as well as age and ambulation status, we could explore these data for correlations with age at loss of ambulation. Using multivariate analysis, current steroid usage was the most significant and largest independent predictor of improved wheelchair-free survival. Thus, these online self-report d

SUBMITTER: Wang RT 

PROVIDER: S-EPMC4207635 | biostudies-literature | 2014 Oct

REPOSITORIES: biostudies-literature