Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study

Abstract Late onset Pompe disease (LOPD) is a slow, progressive disorder characterized by skeletal and respiratory muscle weakness. Enzyme replacement therapy (ERT) slows down the progression of muscle symptoms. Reliable biomarkers are needed to follow up ERT-treated and asymptomatic LOPD patients i...

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Bibliographic Details
Main Authors: Sebastian Figueroa-Bonaparte, Jaume Llauger, Sonia Segovia, Izaskun Belmonte, Irene Pedrosa, Elena Montiel, Paula Montesinos, Javier Sánchez-González, Alicia Alonso-Jiménez, Eduard Gallardo, Isabel Illa, Spanish Pompe group, Jordi Díaz-Manera
Format: Article
Language:English
Published: Nature Portfolio 2018-07-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-018-29170-7