• Gunnar M Buyse, Nathalie Goemans, Marleen van den Hauwe, Daisy Thijs, Imelda J M de Groot, Ulrike Schara, Berten Ceulemans, Thomas Meier, and Luc Mertens.
• Child Neurology, University Hospitals Leuven, Leuven, Belgium. gunnar.buyse@uzleuven.be
• Neuromuscul. Disord. 2011 Jun 1; 21 (6): 396-405.

AbstractEarly 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 8-16 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.Copyright © 2011 Elsevier B.V. All rights reserved.

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