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...
| Main Authors: | , |
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| Format: | Online |
| Language: | English |
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Dove Medical Press
2016
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4913537/ |