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Large scale serum proteomics identifies proteins associated with performance decline and clinical milestones in Duchenne muscular dystrophy
SWME, SVH, and KCHH are employees of BioSymetrics, which has a commercial interest in the results. The remaining authors have no competing interests. This study was funded by Parent Project Muscular Dystrophy through the Protein Mapping Project.
Clinical importance of changes in magnetic resonance biomarkers for Duchenne muscular dystrophy
Introduction Therapeutic development for Duchenne muscular dystrophy (DMD) has progressed rapidly in recent years, with conditional approvals of dystrophin-restoration therapies in both the USA and Europe and myriad potential therapies in preclinical and clinical development.1 However, robust demonstration of therapeutic efficacy has been a challenge in DMD.
Evaluating Genetic Modifiers of Duchenne Muscular Dystrophy Disease Progression Using Modeling and MRI
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