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Title: Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: Results from a 12 month, double-blind, randomized placebo-controlled trial
Authors: Buyse, Gunnar ×
Goemans, Nathalie
Van den Hauwe, Marleen
Thijs, Daisy
de Groot, Imelda J M
Schara, Ulrike
Ceulemans, Berten
Meier, Thomas
Mertens, Luc #
Issue Date: Mar-2011
Publisher: Pergamon Press
Series Title: Neuromuscular Disorders vol:21 pages:396-405
Abstract: 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 8-16years) were randomly assigned to daily treatment with 450mg idebenone (Catena®) (n=13) or placebo (n=8) for 12months. 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.
URI: 
ISSN: 0960-8966
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Brain & Metabolism Section (-)
Section Child - Miscellaneous (-)
Department of Rehabilitation Sciences - miscellaneous
Cardiovascular Imaging and Dynamics
Neuro-musculo-skeletal Research (-)
× corresponding author
# (joint) last author

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