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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Dades bibliogràfiques
Publicat a:Orphanet J Rare Dis
Autors principals: Hendriksz, Christian J., Berger, Kenneth I., Lampe, Christina, Kircher, Susanne G., Orchard, Paul J., Southall, Rebecca, Long, Sarah, Sande, Stephen, Gold, Jeffrey I.
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2016
Matèries:
Review
Accés en línia: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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