Regular ArticleLow-Dose N-Butyldeoxynojirimycin (OGT 918) for Type I Gaucher Disease☆
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Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis
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Cited by (104)
Recommendations for oral treatment for adult patients with type 1 Gaucher disease
2022, Revista Clinica EspanolaSphingolipids and lysosomal pathologies
2014, Biochimica et Biophysica Acta - Molecular and Cell Biology of LipidsGaucher disease and Fabry disease: New markers and insights in pathophysiology for two distinct glycosphingolipidoses
2014, Biochimica et Biophysica Acta - Molecular and Cell Biology of LipidsCitation Excerpt :Several hydrophobic iminosugars inhibit GCS [100–102]. One of them, N-butyldeoxynojirimycin (Zavesca, Actelion), is already a registered drug for oral treatment of Gaucher disease and shown to reverse organomegaly and hematological symptoms in mildly affected type 1 GD patients [103–108]. More potent and specific iminosugar-type inhibitors of GCS, like AMP-DNM (N-(5-adamantane-1-yl-methoxy-pentyl)-deoxynojirimycin) [109], have been designed, as well as ceramide-analogues inhibiting the enzyme [110].
Miglustat therapy in type 1 Gaucher disease: Clinical and safety outcomes in a multicenter retrospective cohort study
2013, Blood Cells, Molecules, and DiseasesCitation Excerpt :Head-to-head clinical trials or comparative effectiveness studies would be needed for such a comparison, and to date no such studies have been reported. Previous clinical trials assessing the efficacy of miglustat in GD1 have primarily been based on changes in liver volume [5,6,9,12], which is viewed as a consistent clinical indicator of disease course and treatment effects [20]. However, in our study only a small number of patients underwent consistent spleen and liver volume assessments, reflecting the fact that organ volumes are not routinely measured in GD1 patients in clinical practice.
Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease
2013, Blood Cells, Molecules, and DiseasesCitation Excerpt :Although outcome measures were globally unchanged after the initial 6 months, there were individual improvements in disease-specific parameters. The concept of a dose-dependency as was seen in the SRT low-dose miglustat trial [31] may also be applicable to PCs. In the low-dose miglustat trial [31] in patients with type 1 Gaucher disease, halving the dose from that which proved to be effective in the seminal trial [32] resulted in halving the efficacy (but the rate and intensity of adverse events were not halved).
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Communicated by E. Beutler, M.D., 02/08/02
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Correspondence and reprint requests to: Deborah Elstein, Gaucher Clinic, Shaare Zedek Medical Center, P.O. Box 3235, Jerusalem 91031, Israel. Fax: +972-2-651-7979. E-mail: [email protected].