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...

Πλήρης περιγραφή

Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Κύριοι συγγραφείς: 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
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: "Paediatrician" Publishers LLC 2018-04-01
Σειρά:Voprosy Sovremennoj Pediatrii
Θέματα:
children
mucopolysaccharidosis
enzyme replacement therapy
laronidase
idursulfase
galsulfase
glycosaminoglycans
Διαθέσιμο Online:https://vsp.spr-journal.ru/jour/article/view/1868
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