Article
The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK.
Orphanet journal of rare diseases - 16 Sept 2008
Moore David, Connock Martin J, Wraith Ed, Lavery Christine
Abstract excerpt
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 understand the prevalence and natural progression of the disease especially with regard to life-expectancy. In...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
