Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis

Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis

Mucopolysaccharidosis type IVA (MPS IVA or Morquio A), a lysosomal storage disease with an autosomal recessive inherited pattern, is induced by <i>GALNS</i> gene mutations causing deficiency in N-acetylgalactosamine-6-sulfatase activity (GALNS; EC 3.1.6.4). Currently, intravenous (IV) en...

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Bibliographic Details
Main Authors: Chung-Lin Lee, Chih-Kuang Chuang, Yu-Min Syu, Huei-Ching Chiu, Yuan-Rong Tu, Yun-Ting Lo, Ya-Hui Chang, Hsiang-Yu Lin, Shuan-Pei Lin
Format: Article
Language:English
Published: MDPI AG 2022-08-01
Series:Journal of Personalized Medicine
Subjects:
mucopolysaccharidosis type IVA
Morquio A
enzyme replacement therapy
elosulfase alfa
meta-analysis
Online Access:https://www.mdpi.com/2075-4426/12/8/1338

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