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

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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Bibliographic Details
Main Authors: Aditi Korlimarla, Jeong-A Lim, Paul McIntosh, Kanecia Zimmerman, Baodong D. Sun, Priya S. Kishnani
Format: Article
Language:English
Published: MDPI AG 2021-07-01
Series:Journal of Clinical Medicine
Subjects:
late-onset Pompe disease
gastrointestinal
smooth muscles
PROMIS–GI symptom scales
GAAKO mice
glycogen storage disorder
Online Access:https://www.mdpi.com/2077-0383/10/15/3395

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https://www.mdpi.com/2077-0383/10/15/3395

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