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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Kaydedildi:
Detaylı Bibliyografya
Yayımlandı:JIMD Rep
Asıl Yazarlar: Fernandes, Samuela A., Khan, Aleena A., Boggs, Tracy, Bowling, Michael, Austin, Stephanie, Stefanescu, Mihaela, Case, Laura, Kishnani, Priya S.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: John Wiley & Sons, Inc. 2020
Konular:
Research Reports
Online Erişim: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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