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Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome

Mucopolysaccharidosis type IVA (MPS IVA or Morquio A) is an autosomal recessive disorder and is one of the lysosomal storage diseases. Patients with MPS IVA have a striking skeletal phenotype but normal intellect. The phenotypic continuum of MPS IVA ranges from severe and rapid progress to mild and...

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Detalles Bibliográficos
Autores principales:	Lee, Chung-Lin, Chuang, Chih-Kuang, Chiu, Huei-Ching, Tu, Ru-Yi, Lo, Yun-Ting, Chang, Ya-Hui, Lin, Shuan-Pei, Lin, Hsiang-Yu
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Dove 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8759989/ https://www.ncbi.nlm.nih.gov/pubmed/35046639 http://dx.doi.org/10.2147/DDDT.S219433

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