Clinical outcomes in elderly patients with Morquio a syndrome receiving enzyme replacement therapy - experience in a Colombian center

INTRODUCTION: Mucopolysaccharidosis type IV A (MPS IVA) or Morquio A syndrome is an autosomal recessive lysosomal storage disease caused by GALNS gene mutations that lead to a deficiency of the N-acetylgalactosamine-6-sulfate sulfatase enzyme and the accumulation of two glycosaminoglycans in cell ly...

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Detalles Bibliográficos
Autores principales: Erazo-Narváez, Andrés Felipe, Muñoz-Vidal, Juan Manuel, Rodríguez-Vélez, Guillermo Hernando, Acosta-Aragón, María Amparo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7718482/
https://www.ncbi.nlm.nih.gov/pubmed/33304816
http://dx.doi.org/10.1016/j.ymgmr.2020.100679

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