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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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| Опубликовано в: : | Orphanet J Rare Dis |
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| Главные авторы: | , , , , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
BioMed Central
2016
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| Предметы: | |
| Online-ссылка: | 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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