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Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy
BACKGROUND: Pompe disease is a rare lysosomal storage disorder characterized by muscle weakness and wasting. The majority of adult patients have slowly progressive disease, which gradually impairs mobility and respiratory function and may lead to wheelchair and ventilator dependency. It is as yet un...
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| Główni autorzy: | , , , , , , |
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| Format: | Artigo |
| Język: | Inglês |
| Wydane: |
BioMed Central
2011
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| Hasła przedmiotowe: | |
| Dostęp online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3135500/ https://ncbi.nlm.nih.gov/pubmed/21631931 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-6-34 |
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