Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease

References

• MehtaAEpidemiology and natural history of Gaucher’s diseaseEur J Intern Med200617S2S517011471
   PubMed Web of Science ®Google Scholar
• HollakCEAn evidence-based review of the potential benefits of taliglucerase alfa in the treatment of patients with Gaucher diseaseCore Evid20127152022654679
   PubMedGoogle Scholar
• Goker-AlpanOTherapeutic approaches to bone pathology in Gaucher disease: past, present and futureMol Genet Metab201110443844721889384
   PubMed Web of Science ®Google Scholar
• ConradiNGKalimoHSouranderPReactions of vessel walls and brain parenchyma to the accumulation of Gaucher cells in the Norrbottnian type (type III) of Gaucher diseaseActa Neuropathol1988753853903364161
   PubMed Web of Science ®Google Scholar
• GrabowskiGAGaucher disease: lessons from a decade of therapyJ Pediatr2004144S15S1915126979
   PubMed Web of Science ®Google Scholar
• BarneveldRAKeijzerWTegelaersFPAssignment of the gene coding for human beta-glucocerebrosidase to the region q21–q31 of chromosome 1 using monoclonal antibodiesHum Genet1983642272316885065
   PubMed Web of Science ®Google Scholar
• AertsJMHollakCBootRGroenerABiochemistry of glycosphingolipid storage disorders: implications for therapeutic interventionPhilos Tran R Soc Lond B Biol Sci2003358905914
   PubMed Web of Science ®Google Scholar
• PastoresGMHughesDAGaucher disease Available from: http://www.ncbi.nlm.nih.gov/books/NBK1269/?reportAccessed June 8, 2015
   Google Scholar
• ZimranAElsteinDLipid storage diseasesLichtmanMAKippsTSeligsohnUKaushanskyKPrchalJTWilliams Hematology8th edNew York, NY, USAMcGraw-Hill2010
   Google Scholar
• LeeJYLeeBHKimGHClinical and genetic characteristics of Gaucher disease according to phenotypic subgroupsKorean J Pediatr201255485322375149
   PubMedGoogle Scholar
• MartinsAMValadaresERPortaGRecommendations on diagnosis, treatment, and monitoring for Gaucher diseaseJ Pediatr2009155S10S1819765407
   PubMed Web of Science ®Google Scholar
• National Gaucher Foundation Inc Available from: http://www.gaucherdisease.org/Accessed June 1, 2015
   Google Scholar
• National Organization of Rare DisordersGaucher disease Available from: http://www.rarediseases.org/rare-disease-information/rare-diseases/byID/12/viewFullReportAccessed June 1, 2015
   Google Scholar
• AltarescuGSchiffmannRParkerCCComparative efficacy of dose regimens in enzyme replacement therapy of type I Gaucher diseaseBlood Cells Mol Dis20002628529011042029
   PubMed Web of Science ®Google Scholar
• No authors listedGaucher disease. Current issues in diagnosis and treatment. NIH Technology Assessment Panel on Gaucher DiseaseJAMA19962755485538606477
   PubMed Web of Science ®Google Scholar
• ZimranAGelbartTWestwoodBGrabowskiGABeutlerEHigh frequency of the Gaucher disease mutation at nucleotide 1226 among Ashkenazi JewsAm J Hum Genet1991498558891897529
   PubMed Web of Science ®Google Scholar
• CoxTMGaucher disease: clinical profile and therapeutic developmentsBiologics2010429921321209725
   PubMedGoogle Scholar
• HarmanciOBayraktarYGaucher disease: new developments in treatment and etiologyWorld J Gastroenterol2008143968397318609679
   PubMed Web of Science ®Google Scholar
• MistryPKCappelliniMDLukinaEA reappraisal of Gaucher disease – diagnosis and disease management algorithmsAm J Hematol20118611011521080341
   PubMed Web of Science ®Google Scholar
• BeightonPGoldblattJSacksSBone involvement in Gaucher diseaseDesnickRGattSGrabowskiGGaucher Disease: A Century of DelineationNew York, NY, USAAlan R Liss1982
   Google Scholar
• BalwaniMFuerstmanLKornreichREdelmannLDesnickRJType 1 Gaucher disease: significant disease manifestations in “asymptomatic” homozygotesArch Intern Med20101701463146920837833
   PubMedGoogle Scholar
• MikoschPGaucher disease and bone: best practice and clinical researchRheumatology201121665681
   Google Scholar
• WineEYanivICohenIJHyper-immunoglobulinemia in pediatric onset type 1 Gaucher disease and effects of enzyme replacementJ Pediatr Hematol Oncol20072945145717609622
   PubMed Web of Science ®Google Scholar
• CapabloJLde CabezónASFraileJAlfonsoPPocoviMGiraldoPNeurological evaluation of patients with Gaucher disease diagnosed as type 1J Neurol Neurosurg Psychiatry20087921922217682016
   PubMed Web of Science ®Google Scholar
• GriffithsGMGaucher disease: forging a new path to the lysosomesCell200713164764918022357
   PubMed Web of Science ®Google Scholar
• DvirHHarelMMcCarthyAAX-ray structure of human acid-beta glucosidase, the defective enzyme in Gaucher diseaseEMBO Rep2003470470912792654
   PubMed Web of Science ®Google Scholar
• KornfeldSStructure and function of the mannose 6-phosphate/insulin like growth factor II receptorsAnnu Rev Biochem1992613073301323236
   PubMed Web of Science ®Google Scholar
• ReczekDSchwakeMSchröderJLIMP-2 is a receptor for lysosomal mannose-6-phosphate-independent targeting of beta glucocerebrosidaseCell200713177078318022370
   PubMed Web of Science ®Google Scholar
• JmoudiakMFutermanAHGaucher disease: pathological mechanisms and modern managementBr J Hematol2005129178188
   PubMed Web of Science ®Google Scholar
• AertsJMKallemeijnWWWegdamWBiomarkers in the diagnosis of lysosomal storage disorders: proteins, lipids, and inhibodiesJ Inherit Metab Dis20113460561921445610
   PubMed Web of Science ®Google Scholar
• BeutlerEKuhlWThe diagnosis of the adult type of Gaucher’s disease and its carrier state by demonstration of deficiency of beta-glucosidase activity in peripheral blood leukocytesJ Lab Clin Med1970767477555477334
   PubMed Web of Science ®Google Scholar
• ZimranAHow I treat Gaucher diseaseBlood20111181463147121670466
   PubMed Web of Science ®Google Scholar
• AertsJMDonker-KoopmanWEvan der VlietMKThe occurrence of two immunologically distinguishable beta glucocerebrosidases in human spleenEur J Biochem19851505655744018098
   PubMedGoogle Scholar
• AertsJMDonker-KoopmanWEKootMBarrangerJATagerJMSchramAWDeficient activity of glucocerebrosidase in urine from patients with type 1 Gaucher diseaseClin Chim Acta19861581551632943536
   PubMed Web of Science ®Google Scholar
• AertsJMHollakCEPlasma and metabolic abnormalities in Gaucher’s diseaseBaillieres Clin Haematol1997106917099497858
   PubMed Web of Science ®Google Scholar
• MoranMTSchofieldJPHaymanARShiGPYoungECoxTMPathologic gene expression in Gaucher disease: upregulation of cysteine proteinases including osteoclastic cathepsin KBlood2000961969197810961902
   PubMed Web of Science ®Google Scholar
• van BreemenMJAertsJMSprengerRRSpeijerDPotential artifacts in proteome analysis of plasma of Gaucher patients due to protease abnormalitiesClin Chim Acta2008396263218640107
   PubMed Web of Science ®Google Scholar
• BarakVAckerMNismanBCytokines in Gaucher’s diseaseEur Cytokine Netw19991020521010400826
   PubMed Web of Science ®Google Scholar
• van BreemenMJde FostMVoermanJSIncreased plasma macrophage inflammatory protein (MIP)-1 alpha and MIP-1 beta levels in type 1 Gaucher diseaseBiochim Biophys Acta2007177278879617499484
   PubMed Web of Science ®Google Scholar
• MollerHJde FrostMAertsHHollakCNoetrupSKPlasma level of macrophage-derived soluble CD163 is increased and positively correlates with severity in Gaucher’s diseaseEur J Haematol20047213513914962251
   PubMed Web of Science ®Google Scholar
• HakalaBEWhiteCReckliesADHuman cartilage gp-39, a major secretory product of articular chondrocytes and synovial cells, is a mammalian member of a chitinase protein familyJ Biol Chem199326825803258108245017
   PubMed Web of Science ®Google Scholar
• OwhashiMAritaHHayaiNIdentification of a novel eosinophil chemotactic cytokine (ECF-L) as a chitinase family proteinJ Biol Chem20002751279128610625674
   PubMed Web of Science ®Google Scholar
• HollakCEMaasMAertsJMClinically relevant therapeutic endpoints in type 1 Gaucher diseaseJ Inherit Metab Dis200124Suppl 29710511758685
   PubMedGoogle Scholar
• DiRoccoMGionaFCarubbiFA new severity score index for phenotypic classification and evaluation of responses to treatment in type I Gaucher diseaseHaematologica2008931211121818603565
   PubMed Web of Science ®Google Scholar
• BootRGVerhoekMde FostMMarked elevation of the chemokine CCL18/PARC in Gaucher disease: a novel surrogate marker for assessing therapeutic interventionsBlood2004103333912969956
   PubMed Web of Science ®Google Scholar
• DeeganBMoranMTMcFarlaneIClinical evaluation of chemokine and enzymatic biomarkers of Gaucher diseaseBlood Cells Mol Dis20053525926716125420
   PubMed Web of Science ®Google Scholar
• BootRGVerhoekMLangeveldMCCL18: a urinary marker of Gaucher cell burden in Gaucher patientsJ Inherit Metab Dis20062956457116736095
   PubMed Web of Science ®Google Scholar
• CoxTMAertsJMBelmatougNManagement of non-neuropathic Gaucher disease with special reference to pregnancy, splenectomy, biphosphonate therapy, use of biomarkers and bone disease monitoringJ Inherit Metab Dis20083131933618509745
   PubMed Web of Science ®Google Scholar
• RolfsAGieseAKGrittnerUGlucosylsphingosine is highly sensitive and specific biomarker for primary diagnostic and follow-up monitoring in Gaucher disease in a non-Jewish, Caucasian cohort of Gaucher disease patientsPLoS One20138e7973224278166
   PubMed Web of Science ®Google Scholar
• http://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=ca02f7a4-ae4f-43c1-a06a-259fe4fcf9cf. Search word: velaglucerase alfaAccessed July 24, 2015
   Google Scholar
• http://dailymed.nlm.gov/dailymed/lookup.cfm? Search word: taliglucerase alfaAccessed December 28, 2012
   Google Scholar
• ShaymanJAEliglustat tartrate: glucosylceramide synthase inhibitor treatment of type 1 Gaucher diseaseDrugs Future20103561362022563139
   PubMed Web of Science ®Google Scholar
• ShaymanJAThe design and clinical development of inhibitors of glycosphingolipid synthesis: will invention be the mother of necessity?Trans Am Clin Climatol Assoc2013124466023874009
   PubMedGoogle Scholar
• BuccolieroRFutermanAHThe roles of ceramide and complex sphingolipids in neuronal cell functionPharmacol Res20034740941912676515
   PubMed Web of Science ®Google Scholar
• http://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=817892d1-ee12-4632-85fc-57ccdf16d7b8. Search word: miglustatAccessed July 24, 2015
   Google Scholar
• SchiffmannRFitzgibbonEJHarrisCRandomized controlled trial of miglustat in Gaucher’s disease type 3Ann Neurol20086451452219067373
   PubMed Web of Science ®Google Scholar
• FutermanAHSussmanJLHorowitzMSilmanIZimranANew directions in the treatment of Gaucher diseaseTrends Pharmacol Sci20042514715115019270
   PubMed Web of Science ®Google Scholar
• FutermanAHHannunYAThe complex life of simple sphingolipidsEMBO Rep2004577778215289826
   PubMed Web of Science ®Google Scholar
• LeeLAbeAShaymanJAImproved inhibitors of glucosylceramide synthaseJ Biol Chem1999274146621466910329660
   PubMed Web of Science ®Google Scholar
• McWachernKAFungJKomarnitskySA specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher diseaseMol Genet Metab20079125926717509920
   PubMed Web of Science ®Google Scholar
• MarshallJMcEachernKAChuangWLImproved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapyJ Inherit Metab Dis20103328128920336375
   PubMed Web of Science ®Google Scholar
• PavlovaEVArcherJWangSZInhibition of UDP-glucosylceramide synthase in mice prevents Gaucher disease-associated B-cell malignancyJ Pathol201523511312425256118
   PubMed Web of Science ®Google Scholar
• CoxTMEliglustat tartrate, an orally glucocerebroside synthase inhibitor for the potential treatment of Gaucher disease and other lysosomal storage diseasesCurr Opin Invest Drugs20101111691181
   PubMed Web of Science ®Google Scholar
• US Food and Drug AdministrationFDA approves new orphan drug to treat a form of Gaucher disease8192014 Available from: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm302549.htmAccessed July 12, 2015
   Google Scholar
• Genzyme CorporationCerdelga (eliglustat) Available from: http://cerdelga.com/pdf/cerdelga_prescribing-information.pdfAccessed June 23, 2015
   Google Scholar
• PooleRMEliglustat: first global approvalDrugs2014741829183625239269
   PubMed Web of Science ®Google Scholar
• PeterschmittMJBurkeABlanksteinLSafety, tolerability, and pharmacokinetics of eliglustat tartrate (Genz-112638) after single dose, multiple doses, and food in healthy volunteersJ Clin Pharmacol20115169570520864621
   PubMed Web of Science ®Google Scholar
• LukinaEWatmanNArreguinEAA Phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1Blood201011689389920439622
   PubMed Web of Science ®Google Scholar
• LukinaEWatmanNArreguinEAImprovement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (GENZ-112638) treatment: 2-year results of a Phase 2 studyBlood20101164095409820713962
   PubMed Web of Science ®Google Scholar
• LukinaEWatmanNDragoskyMEliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatmentBlood Cells Mol Dis20145327427624835462
   PubMed Web of Science ®Google Scholar
• KamathRSLukinaEWatmanNSkeletal improvement in patients with Gaucher disease type 1: a Phase 2 trial of oral eliglustatSkeletal Radiol2014431353136024816856
   PubMed Web of Science ®Google Scholar
• MistryPKLukinaETurkiaHBEffect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1JAMA201531369570625688781
   PubMed Web of Science ®Google Scholar
• CoxTMDrelichmanGCravoREliglustat compared with imiglucerase in patients with Gaucher’s disease type 1 stabilized on enzyme replacement therapy: a Phase 3, randomised, open-label non-inferiority trialLancet20153852355236225819691
   PubMed Web of Science ®Google Scholar
• Genzyme A, Sanofi CompanyA study of eliglustat tartrate (Genz-112638) in patients with Gaucher disease (ENGAGE) Available from: http://www.clinicaltrials.gov/ct2/eliglusatAccessed June 23, 2015
   Google Scholar
• WenstupRJBaileyLGrabowskiGAGaucher disease: alendronate disodium improves bone mineral density in adults receiving enzyme therapyBlood20041041253125715010365
   PubMed Web of Science ®Google Scholar
• SanuelRKatzKPapapoulosSEYosipovitchZZaizovRLibermanUAAmino hydroxy propylidene biphosphonate (APD) treatment improve the clinical skeletal manifestations of Gaucher’s diseasePediatrics1994943853898065868
   PubMed Web of Science ®Google Scholar
• Available from: http://www.goodrx.com/cerdelgaAccessed June 22, 2015
   Google Scholar