Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations

INTRODUCTION: Following a Phase III, randomized, double-blind, placebo (PBO)-controlled, multinational study in subjects with mucopolysaccharidosis IVA (MPS IVA), enzyme replacement therapy (ERT) of elosulfase alfa has been approved in several countries. The study was designed to evaluate safety and...

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Библиографические подробности
Опубликовано в: :Expert Opin Orphan Drugs
Главные авторы: Tomatsu, Shunji, Sawamoto, Kazuki, Shimada, Tsutomu, Bober, Michael B., Kubaski, Francyne, Yasuda, Eriko, Mason, Robert W., Khan, Shaukat, Alméciga-Díaz, Carlos J., Barrera, Luis A., Mackenzie, William G., Orii, Tadao
Формат: Artigo
Язык:Inglês
Опубликовано: 2015
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Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4788508/
https://ncbi.nlm.nih.gov/pubmed/26973801
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1517/21678707.2015.1086640
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