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Role of elosulfase alfa in mucopolysaccharidosis IVA
Mucopolysaccharidosis type IVA (MPS IVA or Morquio A) is an autosomal recessive lysosomal storage disease which results in a striking skeletal phenotype, but does not negatively impact the intellect of the patient. MPS IVA has a phenotypic continuum that ranges from a severe and rapidly progressing...
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| Publicado no: | Appl Clin Genet |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Dove Medical Press
2016
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4913537/ https://ncbi.nlm.nih.gov/pubmed/27366102 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/TACG.S69080 |
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