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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:JIMD Rep
Päätekijät: Fernandes, Samuela A., Khan, Aleena A., Boggs, Tracy, Bowling, Michael, Austin, Stephanie, Stefanescu, Mihaela, Case, Laura, Kishnani, Priya S.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: John Wiley & Sons, Inc. 2020
Aiheet:
Research Reports
Linkit: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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