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- Title
36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy.
- Authors
Regnery, Caroline; Kornblum, Cornelia; Hanisch, Frank; Vielhaber, Stefan; Strigl-Pill, Nicola; Grunert, Birgit; Müller-Felber, Wolfgang; Glocker, Franz Xaver; Spranger, Matthias; Deschauer, Marcus; Mengel, Eugen; Schoser, Benedikt
- Abstract
Glycogen storage disease type 2(GSD2)/Pompe disease is characterized by respiratory and skeletal muscle weakness and atrophy, resulting in functional disability and reduced life span.
- Publication
Journal of inherited metabolic disease, 2012, Vol 35, Issue 5, p837
- ISSN
1573-2665
- Publication type
Journal Article
- DOI
10.1007/s10545-012-9451-8