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...
Main Authors: | , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
"Paediatrician" Publishers LLC
2018-04-01
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Series: | Вопросы современной педиатрии |
Subjects: | |
Online Access: | https://vsp.spr-journal.ru/jour/article/view/1868 |