Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase

Velaglucerase alfa is a glucocerebrosidase produced by gene activation technology in a human fibroblast cell line (HT-1080), and is indicated as an enzyme replacement therapy (ERT) for the treatment of Gaucher disease type 1 (GD1). This multicenter, open-label, 12-month study examined the safety and...

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Autori principali: Zimran, Ari, Pastores, Gregory M., Tylki-Szymanska, Anna, Hughes, Derralynn A., Elstein, Deborah, Mardach, Rebecca, Eng, Christine, Smith, Laurie, Heisel-Kurth, Margaret, Charrow, Joel, Harmatz, Paul, Fernhoff, Paul, Rhead, William, Longo, Nicola, Giraldo, Pilar, Ruiz, Juan A., Zahrieh, David, Crombez, Eric, Grabowski, Gregory A.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2013
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3586535/
https://ncbi.nlm.nih.gov/pubmed/23339116
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajh.23383
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