Timed function tests, motor function measure, and quantitative thigh muscle MRI in ambulant children with Duchenne muscular dystrophy. A cross-sectional analysis

By Simone Schmidt, Patricia Hafner, Andrea Klein, Daniela Rubino-Nacht, Vanya Gocheva, Jonas Schroeder, Arjith Naduvilekoot Devasia, Stephanie Zuesli, Guenther Bernert, Vincent Laugel, Clemens Bloetzer, Maja Steinlin, Andrea Capone, Monika Gloor, Patrick Tobler, Tanja Haas, Oliver Bieri, Thomas Zumbrunn, Dirk Fischer, Ulrike Bonati On Oct 26, 2017

• 6MWD correlates well with MFM total score and its D1 subscore.• Both qMRI values (FF, T2) strongly correlate with clinical assessments.• The qMRI may serve as a biomarker for subclinical changes in DMD-patients.

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A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in duchenne muscular dystrophy

Comorbidities, Clinical Features, and Prognostic Implications of Cancer Patients with Cerebrovascular Disease