EFFECTIVENESS OF MODERN METHODS OF TREATING TYPE I MUCOPOLYSACCHARIDOSIS PATIENTS

• Main Authors: L. S. Namazova-Baranova (Author), N. D. Vashakmadze (Author), M. A. Babaikina (Author), E. N. Basargina (Author), N. V. Zhurkova (Author), A. K. Gevorkyan (Author), L. M. Kuzenkova (Author), T. V. Podkletnova (Author), K. V. Zherdev (Author), O. B. Chelpachenko (Author), T. D. Degtyareva (Author)
• Format: Book
• Published: Union of pediatricians of Russia, 2014-11-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...