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Title
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Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy : results from a 12 month, double-blind, randomized placebo-controlled trial
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Author
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Abstract
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| Early mortality in Duchenne muscular dystrophy (DMD) is related to cardiac and respiratory complications. A phase IIa double-blind randomized placebo-controlled clinical trial was conducted to investigate the tolerability and efficacy of idebenone therapy in children with DMD. Twenty-one DMD patients (aged 816 years) were randomly assigned to daily treatment with 450 mg idebenone (Catena®) (n = 13) or placebo (n = 8) for 12 months. All subjects completed the study and idebenone was safe and well tolerated. Idebenone treatment resulted in a trend (p = 0.067) to increase peak systolic radial strain in the left ventricular inferolateral wall, the region of the heart that is earliest and most severely affected in DMD. A significant respiratory treatment effect on peak expiratory flow was observed (p = 0.039 for PEF and p = 0.042 for PEF percent predicted). Limitations of this study were the small sample size, and a skewed age distribution between treatment groups. Data from this study provided the basis for the planning of a confirmatory study. |
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Language
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English
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Source (journal)
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Neuromuscular disorders
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Publication
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2011
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ISSN
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0960-8966
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DOI
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10.1016/J.NMD.2011.02.016
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Volume/pages
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21
:6
(2011)
, p. 396-405
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ISI
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000291917700003
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Full text (Publisher's DOI)
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