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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Detalhes bibliográficos
Publicado no:JIMD Rep
Main Authors: Fernandes, Samuela A., Khan, Aleena A., Boggs, Tracy, Bowling, Michael, Austin, Stephanie, Stefanescu, Mihaela, Case, Laura, Kishnani, Priya S.
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley & Sons, Inc. 2020
Assuntos:
Research Reports
Acesso em linha: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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