Quantitative whole‐body magnetic resonance imaging in children with Pompe disease: Clinical tools to evaluate severity of muscle disease

OBJECTIVE: Since the introduction of enzyme replacement therapy (ERT) with alglucosidase alfa, there has been increased survival in patients with Pompe disease. It is essential to characterize and quantify the burden of disease in these patients. Here, we report a measure of muscle fat infiltration...

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Publicado en:JIMD Rep
Autores principales: Fernandes, Samuela A., Khan, Aleena A., Boggs, Tracy, Bowling, Michael, Austin, Stephanie, Stefanescu, Mihaela, Case, Laura, Kishnani, Priya S.
Formato: Artigo
Lenguaje:Inglês
Publicado: John Wiley & Sons, Inc. 2020
Materias:
Research Reports
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7802624/
https://ncbi.nlm.nih.gov/pubmed/33473345
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jmd2.12174
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