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The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK

BACKGROUND: Mucopolysaccharidosis type I (MPS I) is a rare lysosomal storage disease subdivided into three phenotypes of increasing severity: Scheie, Hurler-Scheie and Hurler. To gauge the effectiveness of treatments and to determine the load likely to fall on health-care systems, it is necessary to...

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Detalles Bibliográficos
Autores principales: Moore, David, Connock, Martin J, Wraith, Ed, Lavery, Christine
Formato: Texto
Lenguaje:English
Publicado: BioMed Central 2008
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2553763/
https://www.ncbi.nlm.nih.gov/pubmed/18796143
http://dx.doi.org/10.1186/1750-1172-3-24