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This study aims to analyze serum and plasma samples from patients with late onset Pompe disease treated and not treated with enzyme replacement therapy (ERT) to identify microRNA that could be specific of the disease. The investigators will correlate the concentration of these microRNA with several muscle function tests and quantitative muscle MRI to know whether they are good biomarkers of progression.
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35 participants in 2 patient groups
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Jordi Díaz Manera, MD PhD; Eduard Gallardo, PhD
Data sourced from clinicaltrials.gov
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