Journal
NEUROMUSCULAR DISORDERS
Volume 21, Issue 6, Pages 396-405Publisher
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.nmd.2011.02.016
Keywords
Duchenne; Muscular dystrophy; Treatment; Idebenone; Cardiac; Cardiomyopathy; Respiratory function
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Funding
- Santhera Pharmaceuticals
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Early mortality in Duchenne muscular dystrophy (DMD) is related to cardiac and respiratory complications. A phase ha 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 8-16 years) were randomly assigned to daily treatment with 450 mg idebenone (Catena (R)) (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. (C) 2011 Elsevier B.V. All rights reserved.
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