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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Podrobná bibliografie
Vydáno v:Orphanet J Rare Dis
Hlavní autoři: Hendriksz, Christian J., Berger, Kenneth I., Lampe, Christina, Kircher, Susanne G., Orchard, Paul J., Southall, Rebecca, Long, Sarah, Sande, Stephen, Gold, Jeffrey I.
Médium: Artigo
Jazyk:Inglês
Vydáno: BioMed Central 2016
Témata:
Review
On-line přístup: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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