A prospective natural history study of Mucopolysaccharidosis Type IIIA

OBJECTIVES: To characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identified potential endpoints for future treatment trials. STUDY DESIGN: Children with a confirmed diagnosis of MPS IIIA, functioning above a developmental age of 1 year, were followed for up to 2 yea...

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Detalhes bibliográficos
Publicado no:J Pediatr
Main Authors: Shapiro, Elsa G., Nestrasil, Igor, Delaney, Kathleen A., Rudser, Kyle, Kovac, Victor, Nair, Nitin, Richard, Charles W., Haslett, Patrick, Whitley, Chester B.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4769976/
https://ncbi.nlm.nih.gov/pubmed/26787381
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jpeds.2015.11.079
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