טוען...
Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients
BACKGROUND: Pompe disease is a rare metabolic myopathy. In adult patients, progressive weakness of limb-girdle and respiratory muscles often leads to wheelchair and respirator dependency. Clinical studies have shown enzyme replacement therapy (ERT) to positively affect motor and respiratory outcomes...
שמור ב:
| הוצא לאור ב: | Orphanet J Rare Dis |
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| Main Authors: | , , , , , |
| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
BioMed Central
2018
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| נושאים: | |
| גישה מקוונת: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5964954/ https://ncbi.nlm.nih.gov/pubmed/29788986 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0824-4 |
| תגים: |
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