EFFICACY AND SAFETY OF ENZYME REPLACEMENT THERAPY IN CHILDREN WITH MUCOPOLYSACCHARIDOSIS TYPE I, II, AND VI: A SINGLE-CENTER COHORT STUDY

EFFICACY AND SAFETY OF ENZYME REPLACEMENT THERAPY IN CHILDREN WITH MUCOPOLYSACCHARIDOSIS TYPE I, II, AND VI: A SINGLE-CENTER COHORT STUDY

Background. There are limited data on the efficacy of long-term enzyme replacement therapy (ERT) in children with mucopolysaccharidosis (MPS).Objective. Our aim was to study the efficacy and safety of long-term ERT in children with MPS type I, II, and VI.Methods. We analyzed the results of ERT with...

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Main Authors: Liliia A. Osipova (Author), Ludmila M. Kuzenkova (Author), Leyla S. Namazova-Baranova (Author), Anait K. Gevorkyan (Author), Tatiana V. Podkletnova (Author), Nikolay A. Mayanskiy (Author), Grigoriy V. Revunenkov (Author), Nato D. Vashakmadze (Author)
Format: Book
Published: "Paediatrician" Publishers LLC, 2018-04-01T00:00:00Z.
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