Randomized, Controlled Trial of Miglustat in Gaucher’s Disease Type 3

OBJECTIVE: To evaluate the efficacy and safety of miglustat, concomitant with enzyme replacement therapy (ERT), in patients with Gaucher’s disease type 3 (GD3). METHODS: This 24-month, phase II, open-label clinical trial of miglustat in GD3 was conducted in two phases. During the initial 12 months,...

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Detalhes bibliográficos
Main Authors: Schiffmann, Raphael, FitzGibbon, Edmond J., Harris, Chris, DeVile, Catherine, Davies, Elin H., Abel, Larry, van Schaik, Ivo N., Benko, William S., Timmons, Margaret, Ries, Markus, Vellodi, Ashok
Formato: Artigo
Idioma:Inglês
Publicado em: 2008
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2605167/
https://ncbi.nlm.nih.gov/pubmed/19067373
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.21491
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