Clinical characteristics and effects of enzyme replacement therapy with elosulfase alfa in Korean patients with mucopolysaccharidosis type IVA

Mucopolysaccharidosis type IVA (MPS IVA) is a rare autosomal recessive disorder caused by a deficiency in N-acetylgalactosamine-6-sulfatase, which results in skeletal and connective tissue abnormalities, as well as various non-skeletal manifestations. Although enzyme replacement therapy (ERT) is rec...

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Detalles Bibliográficos
Autores principales: Lee, Seung Hoon, Kim, Hwa Young, Cho, Tae-Joon, Kim, Hyoungmin, Ko, Jung Min
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9248211/
https://www.ncbi.nlm.nih.gov/pubmed/35782601
http://dx.doi.org/10.1016/j.ymgmr.2022.100869

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