Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

OBJECTIVE: To assess the efficacy and safety of enzyme replacement therapy (ERT) with BMN 110 (elosulfase alfa) in patients with Morquio A syndrome (mucopolysaccharidosis IVA). METHODS: Patients with Morquio A aged ≥5 years (N = 176) were randomised (1:1:1) to receive elosulfase alfa 2.0 mg/kg/every...

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Detalles Bibliográficos
Autores principales: Hendriksz, Christian J., Burton, Barbara, Fleming, Thomas R., Harmatz, Paul, Hughes, Derralynn, Jones, Simon A., Lin, Shuan-Pei, Mengel, Eugen, Scarpa, Maurizio, Valayannopoulos, Vassili, Giugliani, Roberto, Slasor, Peter, Lounsbury, Debra, Dummer, Wolfgang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Netherlands 2014
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4206772/
https://www.ncbi.nlm.nih.gov/pubmed/24810369
http://dx.doi.org/10.1007/s10545-014-9715-6

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