Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI

OBJECTIVE: To evaluate the efficacy and safety of two dose levels of galsulfase (Naglazyme®) in infants with MPS VI. STUDY DESIGN: This was a phase 4, multicenter, multinational, open-label, two-dose level study. Subjects were randomized 1:1 to receive weekly infusions of 1.0 or 2.0 mg/kg of galsulf...

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Detalles Bibliográficos
Autores principales: Harmatz, Paul R., Garcia, Paula, Guffon, Nathalie, Randolph, Linda M., Shediac, Renée, Braunlin, Elizabeth, Lachman, Ralph S., Decker, Celeste
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Netherlands 2013
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3976509/
https://www.ncbi.nlm.nih.gov/pubmed/24108527
http://dx.doi.org/10.1007/s10545-013-9654-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3976509/
https://www.ncbi.nlm.nih.gov/pubmed/24108527
http://dx.doi.org/10.1007/s10545-013-9654-7

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