טוען...
Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study
BACKGROUND: Pompe disease is a rare metabolic myopathy for which disease-specific enzyme replacement therapy (ERT) has been available since 2006. ERT has shown efficacy concerning muscle strength and pulmonary function in adult patients. However, no data on the effect of ERT on the survival of adult...
שמור ב:
| Main Authors: | , , , , , , , |
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| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
BioMed Central
2013
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| נושאים: | |
| גישה מקוונת: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3623847/ https://ncbi.nlm.nih.gov/pubmed/23531252 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-49 |
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