30 months follow-up of an early enzyme replacement therapy in a severe Morquio A patient: About one case

Patients under 5 years were not evaluated in the phase-3 study for enzyme replacement therapy (ERT) in MPS IV A. Here we describe the evolution of a severe Morquio A pediatric patient who was diagnosed at 19 months old and treated by ERT at 21 months old for the next 30 months. Applying the standard...

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Detalles Bibliográficos
Autores principales: Do Cao, J., Wiedemann, A., Quinaux, T., Battaglia-Hsu, S.F., Mainard, L., Froissart, R., Bonnemains, C., Ragot, S., Leheup, B., Journeau, P., Feillet, F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2016
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5065040/
https://www.ncbi.nlm.nih.gov/pubmed/27761411
http://dx.doi.org/10.1016/j.ymgmr.2016.10.001

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