Outcome Reliability in Non Ambulatory Boys/Men with Duchenne Muscular Dystrophy

BACKGROUND: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. METHODS: Non-ambulatory boys/men with DMD (n=91;16.7± 4.5 years) were assessed by trained clinical evaluators. Feas...

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Detalhes bibliográficos
Publicado no:Muscle Nerve
Main Authors: Connolly, Anne M., Malkus, Elizabeth C., Mendell, Jerry R., Flanigan, Kevin M., Miller, J Philip, Schierbecker, Jeanine R., Siener, Catherine A., Golumbek, Paul T., Zaidman, Craig M., McDonald, Craig M., Johnson, Linda, Nicorici, Alina, Karachunski, Peter I., Day, John W., Kelecic, Jason M., Lowes, Linda P, Alfano, Lindsay N., Darras, Basil T., Kang, Peter B., Quigley, Janet, Pasternak, Amy E., Florence, Julaine M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4305351/
https://ncbi.nlm.nih.gov/pubmed/25056178
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mus.24346
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