Miglustat - CAS 72599-27-0
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Not Intended for Therapeutic Use. For research use only.
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Miglustat is an inhibitor of the ceramide-specific glycosyltransferase, which catalyzes the first step of glycosphingolipid biosynthesis and is currently approved for the oral treatment of type I Gaucher disease (GD1).
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N-Butyldeoxynojirimycin; NB-DNJ; OGT918; Zavesca
1.Prevention of renal damage caused by Shiga toxin type 2: Action of Miglustat on human endothelial and epithelial cells.
Girard MC1, Sacerdoti F1, Rivera FP1, Repetto HA2, Ibarra C1, Amaral MM3. Toxicon. 2015 Oct;105:27-33. doi: 10.1016/j.toxicon.2015.08.021. Epub 2015 Aug 31.
Typical hemolytic uremic syndrome (HUS) is responsible for acute and chronic renal failure in children younger than 5 years old in Argentina. Renal damages have been associated with Shiga toxin type 1 and/or 2 (Stx1, Stx2) produced by Escherichia coli O157:H7, although strains expressing Stx2 are highly prevalent in Argentina. Human glomerular endothelial cells (HGEC) and proximal tubule epithelial cells are very Stx-sensitive since they express high levels of Stx receptor (Gb3). Nowadays, there is no available therapy to protect patients from acute toxin-mediated cellular injury. New strategies have been developed based on the Gb3 biosynthesis inhibition through blocking the enzyme glucosylceramide (GL1) synthase. We assayed the action of a GL1 inhibitor (Miglustat: MG), on the prevention of the renal damage induced by Stx2. HGEC primary cultures and HK-2 cell line were pre-treated with MG and then incubated with Stx2. HK- 2 and HGEC express Gb3 and MG was able to decrease the levels of this receptor.
2.Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients.
Smid BE1, Ferraz MJ2, Verhoek M3, Mirzaian M2, Wisse P4, Overkleeft HS4, Hollak CE1, Aerts JM5,6. Orphanet J Rare Dis. 2016 Mar 24;11(1):28. doi: 10.1186/s13023-016-0413-3.
BACKGROUND: We retrospectively compared biochemical responses in type 1 Gaucher disease patients to treatment with glycosphingolipid synthesis inhibitors miglustat and eliglustat and ERT.
3.Normalisation of brain spectroscopy findings in Niemann-Pick disease type C patients treated with miglustat.
Sedel F1,2, Chabrol B3, Audoin B4, Kaphan E5, Tranchant C6, Burzykowski T7, Tourbah A8, Vanier MT9, Galanaud D10. J Neurol. 2016 Mar 16. [Epub ahead of print]
Niemann-Pick disease type C (NP-C) is a fatal progressive neurolipidosis involving neuronal storage of cholesterol and gangliosides. Miglustat, an inhibitor of glycosphingolipid synthesis, has been approved to treat neurological manifestations in adults and children with NP-C. This open-label observational study in adults with confirmed NP-C evaluated the efficacy of miglustat (200 mg t.i.d.) based on composite functional disability (CFD) scores and brain proton magnetic resonance spectroscopy (H-MRS) measurement of choline (Cho)/N-acetyl aspartate (NAA) ratio in the centrum ovale. Overall, 16 patients were included and received miglustat for a mean period of 30.6 months: 12 continued on miglustat throughout follow up, and 4 discontinued miglustat because of adverse effects (n = 2) or perceived lack of efficacy (n = 2). In the 'continued' subgroup, the mean (SD) annual progression of CFD scores decreased from 0.75 (0.94) before treatment to 0.
4.Urinary excretion and metabolism of miglustat and valproate in patients with Niemann-Pick type C1 disease: One- and two-dimensional solution-state (1)H NMR studies.
Probert F1, Ruiz-Rodado V1, Zhang X2, te Vruchte D3, Claridge TD4, Edgar M5, Tocchio AZ1, Lachmann RH6, Platt FM3, Grootveld M7. J Pharm Biomed Anal. 2016 Jan 5;117:276-88. doi: 10.1016/j.jpba.2015.08.011. Epub 2015 Aug 13.
Niemann-Pick type C1 (NP-C1) disease is a neurodegenerative lysosomal storage disease for which the only approved therapy is miglustat (MGS). In this study we explored the applications and value of both one- and two-dimensional high-resolution NMR analysis strategies to the detection and quantification of MGS and its potential metabolites in urine samples collected from NP-C1 disease patients (n=47), and also applied these techniques to the analysis of the anticonvulsant drug valproate and one of its major metabolites in ca. 30% of these samples (i.e. from those who were also receiving this agent for the control of epileptic seizures). A combination of high-resolution 1D and 2D TOCSY/NOESY techniques confirmed the identity of MGS in the urinary (1)H NMR profiles of NP-C1 patients treated with this agent (n=25), and its quantification was readily achievable via electronic integration of selected 1D resonance intensities. However, this analysis provided little or no evidence for its metabolism in vivo, observations consistent with those acquired in corresponding experiments performed involving an in vitro microsomal system.
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CAS 72599-27-0 Miglustat

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