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Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease
Pompe disease is a lysosomal storage disorder characterized by muscle weakness and cardiomyopathy. It shows a broad variability regarding the clinical severity as well as the age of onset. In the past, two different recombinant enzyme preparations have been developed for the treatment of Pompe patie...
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Formato: | Texto |
Lenguaje: | English |
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Dove Medical Press
2009
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2754091/ https://www.ncbi.nlm.nih.gov/pubmed/19816575 |