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New treatments for the mucopolysaccharidoses: from pathophysiology to therapy
Enzyme replacement therapy is currently considered the standard of care for the treatment of mucopolysaccharidoses (MPS) type I, II, VI, and IV. This approach has shown substantial efficacy mainly on somatic symptoms of the patients, but no benefit was found for other clinical manifestations, such a...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2018
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6238257/ https://www.ncbi.nlm.nih.gov/pubmed/30442204 http://dx.doi.org/10.1186/s13052-018-0564-z |