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...

Descripción completa

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

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9409773/
https://www.ncbi.nlm.nih.gov/pubmed/36013287
http://dx.doi.org/10.3390/jpm12081338

Ejemplares similares