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Health-related quality of life in mucopolysaccharidosis: looking beyond biomedical issues
The mucopolysaccharidoses (MPS) comprise a heterogeneous family of rare, genetic lysosomal storage disorders that result in severe morbidity and reduced life expectancy. Emerging treatments for several of these disorders have triggered the search for clinically relevant biomarkers and clinical marke...
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| 出版年: | Orphanet J Rare Dis |
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| 主要な著者: | , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
BioMed Central
2016
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| 主題: | |
| オンライン・アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5000418/ https://ncbi.nlm.nih.gov/pubmed/27561270 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-016-0503-2 |
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