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Cost-Effectiveness of Enzyme Replacement Therapy (ERT) with Alglucosidase Alfa in Classic-Infantile Patients with Pompe Disease

✍ Scribed by Kanters, T.A.; Plug, I.; Rutten-van Mölken, M.P.M.H.; Redekop, W.; Van der Ploeg, A.T.; Hakkaart, L.


Book ID
122145825
Publisher
John Wiley and Sons
Year
2013
Tongue
English
Weight
60 KB
Volume
16
Category
Article
ISSN
1098-3015

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Morphological changes in muscle tissue o
✍ 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