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A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability

PURPOSE: To evaluate the safety of velaglucerase alfa in patients with type 1 Gaucher disease who received velaglucerase alfa in the US treatment protocol HGT-GCB-058 (ClinicalTrials.gov identifier NCT00954460) during a global supply shortage of imiglucerase. METHODS: This multicenter open-label tre...

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Detalles Bibliográficos
Autores principales: Pastores, Gregory M., Rosenbloom, Barry, Weinreb, Neal, Goker-Alpan, Ozlem, Grabowski, Gregory, Cohn, Gabriel M., Zahrieh, David
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4018500/
https://www.ncbi.nlm.nih.gov/pubmed/24263462
http://dx.doi.org/10.1038/gim.2013.154