Health-Related Quality of Life and Fatigue in Children with Pompe Disease

Health-Related Quality of Life and Fatigue in Children with Pompe Disease

Objective: Pompe disease is an inheritable metabolic myopathy caused by the deficiency of the lysosomal enzyme acid-⍺-glucosidase. The aim of this study was to investigate self-reported and parent-reported health related quality of life (HR-QOL) and fatigue in children with Pompe disease. Study desi...

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Bibliographic Details
Main Authors: Linda E. Scheffers, Karolijn Dulfer, PhD, Charlotte Lanser, Maarten Mackenbach, MD, Ans T. van der Ploeg, MD, PhD, Johanna M.P. van den Hout, MD, PhD, Linda E. van den Berg, MD, PhD
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Journal of Pediatrics: Clinical Practice
Subjects:
Pompe disease
glycogen storage disorder type II
quality of life
fatigue
HR-QOL
Online Access:http://www.sciencedirect.com/science/article/pii/S295054102400022X
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http://www.sciencedirect.com/science/article/pii/S295054102400022X

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