EFFECTIVENESS OF MODERN METHODS OF TREATING TYPE I MUCOPOLYSACCHARIDOSIS PATIENTS

EFFECTIVENESS OF MODERN METHODS OF TREATING TYPE I MUCOPOLYSACCHARIDOSIS PATIENTS

Type I mucopolysaccharidosis (MPS) is a hereditary metabolic disease related to lysosomal storage diseases. Alpha-L-iduronidase enzyme deficiency leads to dissimilar disease phenotypes and varying severity of symptoms. Researchers distinguish between three phenotypes of the disease: Hurler syndrome...

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Main Authors: L. S. Namazova-Baranova, N. D. Vashakmadze, M. A. Babaikina, E. N. Basargina, N. V. Zhurkova, A. K. Gevorkyan, L. M. Kuzenkova, T. V. Podkletnova, K. V. Zherdev, O. B. Chelpachenko, T. D. Degtyareva
Format: Article
Language:Russian
Published: Union of pediatricians of Russia 2014-11-01
Series:Педиатрическая фармакология
Subjects:
mucopolysaccharidosis
bone marrow transplantation
enzyme replacement therapy
carpal tunnel syndrome
dilated cardiomyopathy
Online Access:https://www.pedpharma.ru/jour/article/view/40
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