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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...

詳細記述

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書誌詳細
出版年:Orphanet J Rare Dis
主要な著者: Hendriksz, Christian J., Berger, Kenneth I., Lampe, Christina, Kircher, Susanne G., Orchard, Paul J., Southall, Rebecca, Long, Sarah, Sande, Stephen, Gold, Jeffrey I.
フォーマット: Artigo
言語:Inglês
出版事項: BioMed Central 2016
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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