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The diagnostic journey of patients with mucopolysaccharidosis I: A real-world survey of patient and physician experiences

Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal storage disease. Affected individuals have disease ranging from attenuated to severe with significant disease burden, disability, and premature death. Early treatment with enzyme replacement therapy and/or stem cell transplantation can r...

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Detalles Bibliográficos
Autores principales:	Bruni, Stefano, Lavery, Christine, Broomfield, Alexander
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2016
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4975775/ https://www.ncbi.nlm.nih.gov/pubmed/27536552 http://dx.doi.org/10.1016/j.ymgmr.2016.07.006

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