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New Insights into Gastrointestinal Involvement in Late-Onset Pompe Disease: Lessons Learned from Bench and Bedside

Background: There are new emerging phenotypes in Pompe disease, and studies on smooth muscle pathology are limited. Gastrointestinal (GI) manifestations are poorly understood and underreported in Pompe disease. Methods: To understand the extent and the effects of enzyme replacement therapy (ERT; alg...

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Detalles Bibliográficos
Autores principales: Korlimarla, Aditi, Lim, Jeong-A, McIntosh, Paul, Zimmerman, Kanecia, Sun, Baodong D., Kishnani, Priya S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8347662/
https://www.ncbi.nlm.nih.gov/pubmed/34362174
http://dx.doi.org/10.3390/jcm10153395