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Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy

✍ Scribed by Sean N Prater, Trusha T Patel, Anne F Buckley…

Book ID
120697149
Publisher
BioMed Central
Year
2013
Tongue
English
Weight
575 KB
Volume
8
Category
Article
ISSN
1750-1172

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✍ Léon P. F. Winkel; Joep H. J. Kamphoven; Hannerieke J. M. P. Van Den Hout; Lies 📂 Article 📅 2003 🏛 John Wiley and Sons 🌐 English ⚖ 621 KB

## Abstract Pompe's disease (glycogen storage disease type II) is an autosomal recessive myopathy caused by lysosomal α‐glucosidase deficiency. Enzyme replacement therapy (ERT) is currently under development for this disease. We evaluated the morphological changes in muscle tissue of four children