Role of elosulfase alfa in mucopolysaccharidosis IVA

@inproceedings{Regier2016RoleOE,
  title={Role of elosulfase alfa in mucopolysaccharidosis IVA},
  author={Debra S Regier and Pranoot Tanpaiboon},
  booktitle={The application of clinical genetics},
  year={2016}
}
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 form to a slowly progressive form. The clinical diagnosis is often made in the preschool years based on abnormal bone findings on physical examination and dysplasia on radiographic imaging… CONTINUE READING
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Vimizim (elosulfase alfa) for the treatment of mucopolysaccharidosis type IVA (Morquio syndrome): briefing document for the endocrinologic and metabolic drugs advisory committee

  • Biomarin Pharmaceuticals
  • Available from: http://www.fda.gov/downloads…
  • 2013
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