Review of miglustat for clinical management in Gaucher disease type 1

References

• AbeAInokuchiJJimboMImproved inhibitors of glucosylceramide synthaseJ Biochem199211119161533217
   PubMed Web of Science ®Google Scholar
• AbeAGregorySLeeLReduction of globotriaosylceramide in Fabry disease mice by substrate deprivationJ Clin Invest20001051563710841515
   PubMed Web of Science ®Google Scholar
• AbeARadinNShaymanJAStructural and stereochemical studies of potent inhibitors of glucosylceramide synthase and tumor cell growthJ Lipid Res199536611217775872
   PubMed Web of Science ®Google Scholar
• AbeAWildSLeeWAgents for the treatment of glycosphingolipid storage disordersCurr Drug Metab20012331811513334
   PubMed Web of Science ®Google Scholar
• AmoryJKMullerCHPageSTMiglustat has no apparent effect on spermatogenesis in normal menHum Reprod200722702717067996
   PubMed Web of Science ®Google Scholar
• AnderssonUSmithDJeyakumarMImproved outcome of N-butyl deoxy- galactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff diseaseNeurobiol Dis2004165061515262262
   PubMed Web of Science ®Google Scholar
• BarbourSEdidinMFeldingHarbermannBGlycolipid depletion using a ceramide analogue (PDMP) alters growth, adhesion, and membrane lipid organization in human A431 cellsJ Cell Physiol199215061091537889
   PubMed Web of Science ®Google Scholar
• BartonNWBradyRODambrosiaJMReplacement therapy for inherited enzyme deficiency: macrophage-targeted glucocerebrosidase for Gaucher’s diseaseN Engl J Med19913241464702023606
   PubMed Web of Science ®Google Scholar
• BembiBMarchettiFGuerciVISubstrate reduction therapy in the infantile form of Tay-Sachs diseaseNeurology2006662788016434676
   PubMed Web of Science ®Google Scholar
• BeutlerEGrabowskiGAScriverCRBeaudetALSlyWSChildsBKinzlerKWVogelsteinBGaucher’s diseaseThe Metabolic and Molecular Bases of Inherited Disease19958New YorkMcGraw-Hill264170
   Google Scholar
• CapabloJLFrancoRde CabezonASNeurologic improvement in a type 3 Gaucher disease patient treated with imiglucerase/miglustat combinationEpilepsia2007481406817433057
   PubMed Web of Science ®Google Scholar
• CharrowJDulisseBGrabowskiGAThe effect of enzyme replacement therapy on bone crisis and bone pain in patients with type1 Gaucher diseaseClin Genet2007712051117309642
   PubMed Web of Science ®Google Scholar
• CoxTLachmannRHollakCNovel oral treatment of Gaucher’s disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesisLancet20003551481510801168
   PubMed Web of Science ®Google Scholar
• CoxTMAertsJMAndriaGAdvisory council to the European working group on Gaucher disease The role of the iminosugar N-butyldeoxynojirimycin (Miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statementJ Inherit Metab Dis2003265132614605497
   PubMed Web of Science ®Google Scholar
• ElsteinDDwekAAttiasDA randomized study of OGT 918 as an oral therapy inpatients previously treated with enzyme replacement for type 1 Gaucher disease2002Presented at the Fifth Workshop of the European Working Group on Gaucher DiseasePrague
   Google Scholar
• ElsteinDDwekAAttiasDOral maintenance clinical trial with Miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacementBlood2007110229630117609429
   PubMed Web of Science ®Google Scholar
• ElsteinDGuedaliaJDonigerGMComputerized cognitive testing in patients with type I Gaucher disease: effects of enzyme replacement and substrate reductionGenet Med200571243015714080
   PubMed Web of Science ®Google Scholar
• ElsteinDHollakCAertsJMSustained therapeutic effects of oral Miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher diseaseJ Inherit Metab Dis2004277576615505381
   PubMed Web of Science ®Google Scholar
• [EPAR] European Public Assessment ReportZavesca [Miglustat]2002
   Google Scholar
• Committee for Proprietary Medicinal Products EPARCPMP/3795/02. London, England: The European Agency for the Evaluation of Medicinal Products [online] URL: http://www.emea.eu.int/humandocs/Humans/EPAR/zavesca/zavesca.htm
   Google Scholar
• GiraldoPLatrePPilareAShort-term effect of miglustat in every day clinical use in treatment-naïve or previously treated patients with type 1 Gaucher’s diseaseHeamatologica2006917036
   PubMed Web of Science ®Google Scholar
• GrabowskiGALeslieNWenstrupREnzyme therapy for Gaucher disease: the first 5 yearsBlood Rev199812115339661800
   PubMed Web of Science ®Google Scholar
• HeareTAlpNjPierstmanDASevere endothelial dysfunction in the aorta of a mouse model of Fabry disease; partial prevention by N-butyldeoxynojirimycin treatmentJ Inherit Metab Dis200730798717189993
   PubMed Web of Science ®Google Scholar
• HeitnerRElsteinDAertsJLow-dose N-butyldeoxy- nojirimycin (OGT 918) for type I Gaucher diseaseBlood Cells Mol Dis2002281273312064906
   PubMed Web of Science ®Google Scholar
• PastoresGMBarnettNLKolodnyEHAn open-label, noncomparative study ofmiglustat in type I Gaucher disease: Efficacy and tolerability over 24 months of treatmentClin Ther20052712152716199246
   PubMed Web of Science ®Google Scholar
• PastoresGMElsteinDHrebicekMEffect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studiesClin Ther20072916455417919546
   PubMed Web of Science ®Google Scholar
• PattersonMCVecchioDPradyHMiglustat for treatment of Niemann-Pick C disease: a randomized controlled studyLancet Neurol200767657217689147
   PubMed Web of Science ®Google Scholar
• PlattFMJeyakumarMAnderssonUInhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapyJ Inherit Metab Dis2001242759011405346
   PubMed Web of Science ®Google Scholar
• RadinNSTreatment of Gaucher disease with enzyme inhibitorGlyco J1996131537
   PubMed Web of Science ®Google Scholar
• RadinNSTreating glucosphingolipid disorders by chemotherapy: Use of approved drugs and over-the-counter remediesJ Inherit Metab Dis2000237677711196103
   PubMed Web of Science ®Google Scholar
• TierneyMPottageJKesslerHThe tolerability and pharmacokinetics of N-butyl-deoxynoirimycin in patients with advanced HIV disease (ACTG100). The AIDS Clinical Trials Group (ACTG) of the National Institute of Allergy and Infectious DiseasesAcquir Immune Defic Syndr Hum Retrovirol19951054953
   Web of Science ®Google Scholar
• US FDA Medical Review2003 [online] URL: http://www.fda.gov/cder/foi/nda/2003/21-348_Zavesca.htm
   Google Scholar
• WaldenCMButtersTDDwekRALong-term non-hormonal male contraception in mice using N-butyldeoxynojirimycinHum Reprod20062113091516396932
   PubMed Web of Science ®Google Scholar
• WeinrebNJCharrowJAnderssonHCEffectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher RegistryAm J Med20021131121912133749
   PubMed Web of Science ®Google Scholar
• YamGHZuberCRothJA synthetic chaperone corrects the trafficking defect and disease phenotype in a protein misfolding disorderFASEB J200519121815629890
   PubMed Web of Science ®Google Scholar
• ZimranAElsteinDGaucher disease and the clinical experience with substrate reduction therapyPhilos Trans R Soc Lond B Biol Sci2003358961612803930
   PubMed Web of Science ®Google Scholar