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38. Quality-of-life changes in MPS II patients receiving enzyme replacement therapy

✍ Scribed by Susan Hale; J. Lawrence Merritt II; Sihoun Hahn; Penny Schubert; Michael Raff

Book ID
116988223
Publisher
Elsevier Science
Year
2008
Tongue
English
Weight
58 KB
Volume
93
Category
Article
ISSN
1096-7192

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Morphological changes in muscle tissue o
Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy
✍ LΓ©on P. F. Winkel; Joep H. J. Kamphoven; Hannerieke J. M. P. Van Den Hout; Lies πŸ“‚ Article πŸ“… 2003 πŸ› John Wiley and Sons 🌐 English βš– 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