83
Views
3
CrossRef citations to date
0
Altmetric
Review

Spotlight on taliglucerase alfa in the treatment of pediatric patients with type 1 Gaucher disease

&
Pages 73-81 | Published online: 16 Jun 2017

References

  • GrabowskiGAPetskoGAKolodnyEHGaucher diseaseScriverCRBeudetALSlyWSThe Metabolic and Molecular Basis of Inherited DiseaseNew York, NYMcGraw-Hill20101143
  • MeiklePJHopwoodJJClagueAECareyWFPrevalence of lysosomal storage disordersJAMA19992812492549918480
  • ZimranAGonzalez-RodriguezDEAbrahamovASafety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher diseaseBlood Cells Mol Dis201554191625453586
  • ZimranAElsteinDLipid storage diseasesLichtmanMAKippsTSeligsohnUKaushanskyKPrchalJTWilliams Hematolog8th edNew York, NYMcGraw-Hill201010651071
  • MistryPGermainDPPhenotype variations in Gaucher diseaseRev Med Interne200627S3S1016644399
  • AnderssonHKaplanPKacenaKYeeJEight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1Pediatrics20081221182119019047232
  • KauliRZaizovRLazarLDelayed growth and puberty in patients with Gaucher disease type 1: natural history and effect of splenectomy and/or enzyme replacement therapyIsr Med Assoc J200015816310804944
  • CharrowJAnderssonHCKaplanPThe Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher diseaseArch Intern Med20001602835284311025794
  • CharrowJAnderssonHCKaplanPEnzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendationsJ Pediatr200414411212014722528
  • BartonNWBradyRODambrosiaJMReplacement therapy for inherited enzyme deficiency—macrophage-targeted glucocerebrosidase for Gaucher’s diseaseN Engl J Med1991324146414702023606
  • Cerezyme [package insert]Cambridge, MAGenzyme Corporation2009
  • VPRIV [package insert]Lexington, MAShire Human Genetic Therapies, Inc.2013
  • Elelyso [package insert]New York, NYPfizer Labs2014
  • ShaaltielYBartfeldDHashmueliSProduction of gluco-cerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher’s disease using a plant cell systemPlant Biotechnol J2007557959017524049
  • FoxJLFirst plant-made biologic approvedNat Biotechnol201230472
  • OparA‘Pharmers’ hope for first plant drug harvestNat Rev Drug Discov201110818221283091
  • AviezerDBrill-AlmonEShaaltielYA plant-derived recombinant human glucocerebrosidase enzyme—a preclinical and phase I investigationPLoS One20094e479219277123
  • GrabowskiGAGolemboMShaaltielYTaliglucerase alfa: an enzyme replacement therapy using pla nt cell expression technologyMol Genet Metab20141121824630271
  • ClinicalTrials.gov, National Institutes of HealthPlant cell expressed recombinant human glucocerebrosidase extension trial NLM identifier: NCT00705939
  • ClinicalTrials.gov, National Institutes of HealthExpanded access trial of plant expressed recombinant glucocerebrosidase (prGCD) in patients with Gaucher disease NLM identifier: NCT00962260
  • ZimranABrill-AlmonEChertkoffRPivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher diseaseBlood20111185767577321900191
  • ZimranAGonzalez-RodriguezDEAbrahamovALong-term safety and efficacy of taliglucerase alfa in pediatric patients with Gaucher disease who were treatment-naïve previously treated with immiglucerase. Abstract 293Mol Genet Metab20151142S129
  • PastoresGMPetakovMGiraldoPA Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imigluceraseBlood Cells Mol Dis201453425326024950666
  • ClinicalTrials.gov, National Institutes of HealthA multicenter extension study of taliglucerase alfa in adult subjects with Gaucher disease NLM identifier: NCT01422187
  • ZimranAGonzalez-RodriguezDEAbrahamovALong-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imigluceraseBlood Cells Mol Dis2016S10799796163022130222
  • ClinicalTrials.gov, National Institutes of HealthA multicenter extension study of taliglucerase alfa in pediatric subjects with Gaucher disease NLM identifier: NCT01411228
  • HughesDAGonzalezDELukinaEAVelaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: long-term data from phase III clinical trialsAm J Hematol201590758459125801797