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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Detaylı Bibliyografya
Yayımlandı:Orphanet J Rare Dis
Asıl Yazarlar: Hendriksz, Christian J., Berger, Kenneth I., Lampe, Christina, Kircher, Susanne G., Orchard, Paul J., Southall, Rebecca, Long, Sarah, Sande, Stephen, Gold, Jeffrey I.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: BioMed Central 2016
Konular:
Review
Online Erişim: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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