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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 GALNS gene mutations causing deficiency in N-acetylgalactosamine-6-sulfatase activity (GALNS; EC 3.1.6.4). Currently, intravenous (IV) enzyme replacement th...

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Detalles Bibliográficos
Autores principales:	Lee, Chung-Lin, Chuang, Chih-Kuang, Syu, Yu-Min, Chiu, Huei-Ching, Tu, Yuan-Rong, Lo, Yun-Ting, Chang, Ya-Hui, Lin, Hsiang-Yu, Lin, Shuan-Pei
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9409773/ https://www.ncbi.nlm.nih.gov/pubmed/36013287 http://dx.doi.org/10.3390/jpm12081338

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