Online Self-Report Data for Duchenne Muscular Dystrophy Confirms Natural History and Can Be Used to Assess for Therapeutic Benefits

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...

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Detalles Bibliográficos
Autores principales: Wang, Richard T., Silverstein Fadlon, Cheri A., Ulm, J. Wes, Jankovic, Ivana, Eskin, Ascia, Lu, Ake, Rangel Miller, Vanessa, Cantor, Rita M., Li, Ning, Elashoff, Robert, Martin, Anne S., Peay, Holly L., Halnon, Nancy, Nelson, Stanley F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2014
Materias:
Natural History and Outcome Measures
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4207635/
https://www.ncbi.nlm.nih.gov/pubmed/25635234
http://dx.doi.org/10.1371/currents.md.e1e8f2be7c949f9ffe81ec6fca1cce6a

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4207635/
https://www.ncbi.nlm.nih.gov/pubmed/25635234
http://dx.doi.org/10.1371/currents.md.e1e8f2be7c949f9ffe81ec6fca1cce6a

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