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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Détails bibliographiques
Auteurs principaux: Liliia A. Osipova, Ludmila M. Kuzenkova, Leyla S. Namazova-Baranova, Anait K. Gevorkyan, Tatiana V. Podkletnova, Nikolay A. Mayanskiy, Grigoriy V. Revunenkov, Nato D. Vashakmadze
Format: Artigo
Langue:Inglês
Publié: "Paediatrician" Publishers LLC 2018-04-01
Collection:Voprosy Sovremennoj Pediatrii
Sujets:
children
mucopolysaccharidosis
enzyme replacement therapy
laronidase
idursulfase
galsulfase
glycosaminoglycans
Accès en ligne:https://vsp.spr-journal.ru/jour/article/view/1868
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