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The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?
Pompe disease/glycogen storage disease type II, is a rare, lysosomal storage disorder associated with progressive proximal myopathy, causing a gradual loss of muscular function and respiratory insufficiency. Studies of patients with late-onset Pompe disease have used endpoints such as the 6-minute w...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2013
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4015278/ https://www.ncbi.nlm.nih.gov/pubmed/24119230 http://dx.doi.org/10.1186/1750-1172-8-160 |