메뉴 건너뛰기




Volumn 12, Issue 1, 2017, Pages

Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: Safety, tolerability, and pharmacokinetics in healthy subjects

Author keywords

Lucerastat; Pharmacokinetics; Safety; Tolerability

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; LUCERASTAT; PLACEBO; 1 DEOXYNOJIRIMYCIN; CARBOHYDRATE; MIGALASTAT;

EID: 85009493971     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-017-0565-9     Document Type: Article
Times cited : (38)

References (21)
  • 1
    • 84892755879 scopus 로고    scopus 로고
    • Lysosomal storage disorders: Old diseases, present and future challenges
    • 24380123
    • Klein AD, Futerman AH. Lysosomal storage disorders: old diseases, present and future challenges. Pediatr Endocrinol Rev. 2013;11 Suppl 1:59-63.
    • (2013) Pediatr Endocrinol Rev , vol.11 , pp. 59-63
    • Klein, A.D.1    Futerman, A.H.2
  • 3
    • 84871603938 scopus 로고    scopus 로고
    • Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: A prospective, open-label non-inferiority study
    • Miglustat Maintenance Study G 10.1186/1750-1172-7-102 23270487 3552937
    • Cox TM, Amato D, Hollak CE, Luzy C, Silkey M, Giorgino R, Steiner RD, Miglustat Maintenance Study G. Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study. Orphanet J Rare Dis. 2012;7:102. doi: 10.1186/1750-1172-7-102.
    • (2012) Orphanet J Rare Dis , vol.7 , pp. 102
    • Cox, T.M.1    Amato, D.2    Hollak, C.E.3    Luzy, C.4    Silkey, M.5    Giorgino, R.6    Steiner, R.D.7
  • 6
    • 84942506112 scopus 로고    scopus 로고
    • Eliglustat: A review in Gaucher disease type 1
    • 10.1007/s40265-015-0468-9 1:CAS:528:DC%2BC2MXhsFCis77J 26384672
    • Scott LJ. Eliglustat: a review in Gaucher disease type 1. Drugs. 2015;75(14):1669-78. doi: 10.1007/s40265-015-0468-9.
    • (2015) Drugs , vol.75 , Issue.14 , pp. 1669-1678
    • Scott, L.J.1
  • 7
    • 0034002146 scopus 로고    scopus 로고
    • Molecular requirements of imino sugars for the selective control of N-linked glycosylation and glycosphingolipid biosynthesis
    • 1:CAS:528:DC%2BD3cXhvVequ7c%3D http://dx.doi.org/10.1016/S0957-4166(99)00468-1
    • Butters TD, van den Broek LAGM, Fleet GWJ, Krulle TM, Wormald MR, Dwek RA, Platt FM. Molecular requirements of imino sugars for the selective control of N-linked glycosylation and glycosphingolipid biosynthesis. Tetrahedron Asymmetry. 2000;11(1):113-24. http://dx.doi.org/10.1016/S0957-4166(99)00468-1.
    • (2000) Tetrahedron Asymmetry , vol.11 , Issue.1 , pp. 113-124
    • Butters, T.D.1    Van Den Broek, L.A.G.M.2    Fleet, G.W.J.3    Krulle, T.M.4    Wormald, M.R.5    Dwek, R.A.6    Platt, F.M.7
  • 9
    • 0028171083 scopus 로고
    • N-butyldeoxygalactonojirimycin inhibits glycolipid biosynthesis but does not affect N-linked oligosaccharide processing
    • 1:CAS:528:DyaK2cXmt1Wjt7k%3D 7929454
    • Platt FM, Neises GR, Karlsson GB, Dwek RA, Butters TD. N-butyldeoxygalactonojirimycin inhibits glycolipid biosynthesis but does not affect N-linked oligosaccharide processing. J Biol Chem. 1994;269(43):27108-14.
    • (1994) J Biol Chem , vol.269 , Issue.43 , pp. 27108-27114
    • Platt, F.M.1    Neises, G.R.2    Karlsson, G.B.3    Dwek, R.A.4    Butters, T.D.5
  • 10
    • 0033952264 scopus 로고    scopus 로고
    • N-butyldeoxygalactonojirimycin: A more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo
    • 1:CAS:528:DC%2BD3cXpsVWmsQ%3D%3D 10718340
    • Andersson U, Butters TD, Dwek RA, Platt FM. N-butyldeoxygalactonojirimycin: a more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo. Biochem Pharmacol. 2000;59(7):821-9.
    • (2000) Biochem Pharmacol , vol.59 , Issue.7 , pp. 821-829
    • Andersson, U.1    Butters, T.D.2    Dwek, R.A.3    Platt, F.M.4
  • 11
    • 0042330085 scopus 로고    scopus 로고
    • Membrane disruption and cytotoxicity of hydrophobic N-alkylated imino sugars is independent of the inhibition of protein and lipid glycosylation
    • 10.1042/BJ20030348 1:CAS:528:DC%2BD3sXms1Gks74%3D 12769816 1223602
    • Mellor HR, Platt FM, Dwek RA, Butters TD. Membrane disruption and cytotoxicity of hydrophobic N-alkylated imino sugars is independent of the inhibition of protein and lipid glycosylation. Biochem J. 2003;374(Pt 2):307-14. doi: 10.1042/BJ20030348.
    • (2003) Biochem J , vol.374 , pp. 307-314
    • Mellor, H.R.1    Platt, F.M.2    Dwek, R.A.3    Butters, T.D.4
  • 12
    • 3242681806 scopus 로고    scopus 로고
    • Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease
    • 10.1016/j.nbd.2004.04.012 1:CAS:528:DC%2BD2cXlslOqu74%3D 15262262
    • Andersson U, Smith D, Jeyakumar M, Butters TD, Borja MC, Dwek RA, Platt FM. Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease. Neurobiol Dis. 2004;16(3):506-15. doi: 10.1016/j.nbd.2004.04.012.
    • (2004) Neurobiol Dis , vol.16 , Issue.3 , pp. 506-515
    • Andersson, U.1    Smith, D.2    Jeyakumar, M.3    Butters, T.D.4    Borja, M.C.5    Dwek, R.A.6    Platt, F.M.7
  • 13
    • 40949120522 scopus 로고    scopus 로고
    • N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice
    • 10.1016/j.neuint.2007.12.001 1:CAS:528:DC%2BD1cXktVKls7c%3D 18207611
    • Baek RC, Kasperzyk JL, Platt FM, Seyfried TN. N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice. Neurochem Int. 2008;52(6):1125-33. doi: 10.1016/j.neuint.2007.12.001.
    • (2008) Neurochem Int , vol.52 , Issue.6 , pp. 1125-1133
    • Baek, R.C.1    Kasperzyk, J.L.2    Platt, F.M.3    Seyfried, T.N.4
  • 15
    • 84937206815 scopus 로고    scopus 로고
    • A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat
    • 10.1186/s13023-015-0297-7 26084276 4501118
    • Remenova T, Morand O, Amato D, Chadha-Boreham H, Tsurutani S, Marquardt T. A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat. Orphanet J Rare Dis. 2015;10:81. doi: 10.1186/s13023-015-0297-7.
    • (2015) Orphanet J Rare Dis , vol.10 , pp. 81
    • Remenova, T.1    Morand, O.2    Amato, D.3    Chadha-Boreham, H.4    Tsurutani, S.5    Marquardt, T.6
  • 16
    • 84973759313 scopus 로고
    • Assessment of dose proportionality: Report from the statisticians in the pharmaceutical industry/pharmacokinetics UK joint working party
    • Gough K, Hutchison M, Keene O, Byrom B, Ellis S, Lacey L, McKellar J. Assessment of dose proportionality: report from the statisticians in the pharmaceutical industry/pharmacokinetics UK joint working party. Drug Inf J. 1995;29(3):1039-48. doi: 10.1177/009286159502900324.
    • (1995) Drug Inf J , vol.29 , Issue.3 , pp. 1039-1048
    • Gough, K.1    Hutchison, M.2    Keene, O.3    Byrom, B.4    Ellis, S.5    Lacey, L.6    McKellar, J.7
  • 18
    • 13444282229 scopus 로고    scopus 로고
    • Liver enzyme alteration: A guide for clinicians
    • 10.1503/cmaj.1040752 15684121 545762
    • Giannini EG, Testa R, Savarino V. Liver enzyme alteration: a guide for clinicians. CMAJ. 2005;172(3):367-79. doi: 10.1503/cmaj.1040752.
    • (2005) CMAJ , vol.172 , Issue.3 , pp. 367-379
    • Giannini, E.G.1    Testa, R.2    Savarino, V.3
  • 19
    • 0033061022 scopus 로고    scopus 로고
    • Transaminase elevation on placebo during phase i trials: Prevalence and significance
    • 10.1046/j.1365-2125.1999.00952.x 1:STN:280:DyaK1MzhsFaqsw%3D%3D 10383555 2014876
    • Rosenzweig P, Miget N, Brohier S. Transaminase elevation on placebo during phase I trials: prevalence and significance. Br J Clin Pharmacol. 1999;48(1):19-23. doi: 10.1046/j.1365-2125.1999.00952.x.
    • (1999) Br J Clin Pharmacol , vol.48 , Issue.1 , pp. 19-23
    • Rosenzweig, P.1    Miget, N.2    Brohier, S.3
  • 20
    • 33947314170 scopus 로고    scopus 로고
    • The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat
    • 10.1080/00498250601094543 1:CAS:528:DC%2BD2sXltFGjtbc%3D 17624027
    • Treiber A, Morand O, Clozel M. The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat. Xenobiotica. 2007;37(3):298-314. doi: 10.1080/00498250601094543.
    • (2007) Xenobiotica , vol.37 , Issue.3 , pp. 298-314
    • Treiber, A.1    Morand, O.2    Clozel, M.3
  • 21
    • 85009489249 scopus 로고    scopus 로고
    • Lucerastat, an iminosugar for substrate reduction therapy: Safety, tolerability, pharmacodynamics, and pharmacokinetics in adult subjects with Fabry disease
    • Abstract number 17-A-247
    • Guérard N, Oder D, Nordbeck P, Zwingelstein C, Morand O, Welford R, Dingemanse J, Wanner C. Lucerastat, an iminosugar for substrate reduction therapy: safety, tolerability, pharmacodynamics, and pharmacokinetics in adult subjects with Fabry disease. WORLDSymposium, Abstract number 17-A-247. 2017.
    • (2017) WORLDSymposium
    • Guérard, N.1    Oder, D.2    Nordbeck, P.3    Zwingelstein, C.4    Morand, O.5    Welford, R.6    Dingemanse, J.7    Wanner, C.8


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.