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

• Principais autores: Liliia A. Osipova (Autor), Ludmila M. Kuzenkova (Autor), Leyla S. Namazova-Baranova (Autor), Anait K. Gevorkyan (Autor), Tatiana V. Podkletnova (Autor), Nikolay A. Mayanskiy (Autor), Grigoriy V. Revunenkov (Autor), Nato D. Vashakmadze (Autor)
• Formato: Livro
• Publicado em: "Paediatrician" Publishers LLC, 2018-04-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...