Elosulfase alfa for mucopolysaccharidosis type IVA: Real-world experience in 7 patients from the Spanish Morquio-A early access program

There is a growing interest in evaluating the effectiveness of enzyme replacement therapy (ERT) with elosulfase alfa in patients with mucopolysaccharidosis type IVA (MPS-IVA) under real-world conditions. We present the experience of seven pediatric MPS-IVA patients from the Spanish Morquio-A Early A...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab Rep
Main Authors: Pintos-Morell, Guillem, Blasco-Alonso, Javier, Couce, María L., Gutiérrez-Solana, Luís G., Guillén-Navarro, Encarna, O'Callaghan, Mar, del Toro, Mireia
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2018
Assuntos:
Research Paper
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6047108/
https://ncbi.nlm.nih.gov/pubmed/30023300
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2018.03.009
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