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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Detalles Bibliográficos
Autores principales: Hendriksz, Christian J., Berger, Kenneth I., Lampe, Christina, Kircher, Susanne G., Orchard, Paul J., Southall, Rebecca, Long, Sarah, Sande, Stephen, Gold, Jeffrey I.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2016
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5000418/
https://www.ncbi.nlm.nih.gov/pubmed/27561270
http://dx.doi.org/10.1186/s13023-016-0503-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5000418/
https://www.ncbi.nlm.nih.gov/pubmed/27561270
http://dx.doi.org/10.1186/s13023-016-0503-2

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