Article
Short-term effect of miglustat in every day clinical use in treatment-naïve or previously treated patients with type 1 Gaucher's disease.
Haematologica - 1 May 2006
Giraldo Pilar, Latre Paz, Alfonso Pilar, Acedo Antonio, Alonso Dora, Barez Abelardo, Corrales Alejandro, Franco Rafael, Roldan Vanessa, Serrano Sol, Pocovi Miguel
Abstract excerpt
In a prospective, open-label study, 25 patients with mild-to-moderate type 1 Gaucher's disease (GD1) were treated with miglustat (Zavesca), an oral glucosylceramide synthase inhibitor, over 12 months. Of the 25 patients, 10 were therapy-naïve and 15 had previously received enzyme replacement therapy (ERT). Clinical status, blood parameters, biomarkers, and organomegaly were assessed at baseline at 6 months and at...
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