Quantitative muscle MRI: A powerful surrogate outcome measure in Duchenne muscular dystrophy

Ulrike Bonati,Patricia Hafner,Sabine Schädelin,Maurice Schmid,Arjith Naduvilekoot Devasia,Jonas Schroeder,Stephanie Zuesli,Urs Pohlman,Cornelia Neuhaus,Andrea Klein,Michael Sinnreich,Tanja Haas,Monika Gloor,Oliver Bieri,Arne Fischmann,Dirk Fischer

Neuromuscular Disorders（2015）

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摘要

•Powerful outcome measures for clinical trials in neuromuscular disorders are needed.•In this study the effect size of qMRI was greater than that of physical scales (MFM).•Sample size estimations for qMRI were up to 17 fold smaller compared to the MFM.•QMRI is safe, independent of ambulatory abilities and well tolerated by children.

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关键词

Duchenne muscular dystrophy,Outcome measure,Endpoint,Quantitative MRI,Neuromuscular disorders

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