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...

وصف كامل

التفاصيل البيبلوغرافية
الحاوية / القاعدة:Вопросы современной педиатрии
المؤلفون الرئيسيون: 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
التنسيق: مقال
اللغة:الإنجليزية
منشور في: "Paediatrician" Publishers LLC 2018-04-01
الموضوعات:
children
mucopolysaccharidosis
enzyme replacement therapy
laronidase
idursulfase
galsulfase
glycosaminoglycans
الوصول للمادة أونلاين:https://vsp.spr-journal.ru/jour/article/view/1868

الانترنت

https://vsp.spr-journal.ru/jour/article/view/1868

مواد مشابهة