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

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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Autores principales: 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
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2018
Materias:
Medicine
R
Science
Q
Acceso en línea:https://doaj.org/article/92acffe4ea3d495381a7f63dd8c21825
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