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Mucopolysaccharidosis I; Parental beliefs about the impact of disease on the quality of life of their children
BACKGROUND: Hematopoietic stem cell transplants, alongside enzyme replacement therapy and good multi-disciplinary care, have dramatically improved the life expectancy in children with Mucopolysaccharidosis (MPS) I, with better objective and functional outcomes. Despite these improvements, children w...
Autores principales: | Soni-Jaiswal, A., Mercer, J., Jones, S. A., Bruce, I. A., Callery, P. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4942895/ https://www.ncbi.nlm.nih.gov/pubmed/27406185 http://dx.doi.org/10.1186/s13023-016-0478-z |
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