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Mucopolysaccharidosis type hurler-scheie



Mucopolysaccharidosis type hurler-scheie Causes


The disease is brought about by a deficiency of the lysosomal enzyme a-L-iduronidase. Accumulation of the undergraded mucopolysaccharides DS and HS in tissues and organs and storage of excess mucopolysaccharides also causes the condition.


Mucopolysaccharidosis type hurler-scheie Definition


Hurler-Scheie is a subtype of MPS I, along with the Hurler syndrome. It is a deficiency in the a-L-iduronidase enzyme and the lysosomal accumulation of mucopolysaccharides.


Mucopolysaccharidosis type hurler-scheie Diagnosis


Clinical examination and conducting enzyme assays can provide definitive diagnosis of the disease.


Mucopolysaccharidosis type hurler-scheie Symptoms and Signs


The symptoms of the disease onset vary considerably both within and among the 3 subgroups. Its clinical features actually lie between those of Hurler and Scheie syndrome. Just like the other subtypes, progressive involvement of the various organs and tissues of the body occur.


Mucopolysaccharidosis type hurler-scheie Treatment


Medical care for the disease includes enzyme replacement therapy with laronidase. It may improve pulmonary function and walking ability and reduction of excess carbohydrates stored in organs.


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