Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y

BACKGROUND: Previous studies have shown that elosulfase alfa has a favorable efficacy/safety profile in Morquio A patients aged ≥5 y. This study evaluated safety and impact on urine keratan sulfate (uKS) levels and growth velocity in younger patients. METHODS: Fifteen Morquio A patients aged <5 y...

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Detalhes bibliográficos
Publicado no:Pediatr Res
Main Authors: Jones, Simon A., Bialer, Martin, Parini, Rossella, Martin, Ken, Wang, Hui, Yang, Ke, Shaywitz, Adam J., Harmatz, Paul
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
Assuntos:
Clinical Investigation
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4700045/
https://ncbi.nlm.nih.gov/pubmed/26331768
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/pr.2015.169
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