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Improvement in Cardiac Function With Enzyme Replacement Therapy in a Patient With Infantile-Onset Pompe Disease
BACKGROUND: Pompe disease is a lysosomal storage disorder that results from an inborn error of metabolism involving abnormal glycogen storage. Infantile-onset Pompe disease is the most severe phenotype, and enzyme replacement therapy with alglucosidase alfa (Lumizyme) improves medical and functional...
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| Pubblicato in: | Ochsner J |
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| Autori principali: | , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Academic Division of Ochsner Clinic Foundation
2018
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6292475/ https://ncbi.nlm.nih.gov/pubmed/30559630 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.31486/toj.18.0049 |
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