메뉴 건너뛰기




Volumn 34, Issue 1, 2009, Pages 23-25

Isofagomine tartrate: Glycogen phosphorylase inhibitor treatment of Gaucher's disease

Author keywords

[No Author keywords available]

Indexed keywords

AT 2101; ENZYME INHIBITOR; GLUCOSYLCERAMIDASE; GLUCOSYLCERAMIDE; GLYCOGEN PHOSPHORYLASE; HGT 3410; ISOFAGOMINE TARTRATE; NN 42 1007; PLICERA; UNCLASSIFIED DRUG;

EID: 76649093920     PISSN: 03778282     EISSN: None     Source Type: Journal    
DOI: 10.1358/dof.2009.034.01.1323946     Document Type: Review
Times cited : (12)

References (35)
  • 1
    • 0034605845 scopus 로고    scopus 로고
    • A general strategy towards the synthesis of 1-N-iminosugar type glycosidase inhibitors: Demonstration by the synthesis of D-as well as L-glucose type iminosugars (isofagomines)
    • Pandey, G., Kapur, M. A general strategy towards the synthesis of 1-N-iminosugar type glycosidase inhibitors: Demonstration by the synthesis of D-as well as L-glucose type iminosugars (isofagomines). Tetrahedron Lett 2000, 41(45): 8821-4.
    • (2000) Tetrahedron Lett , vol.41 , Issue.45 , pp. 8821-8824
    • Pandey, G.1    Kapur, M.2
  • 2
    • 0023250442 scopus 로고
    • Total synthesis of (+)-nojirimycin and (+)-1-deoxynojirimycin
    • Iida, H. et al. Total synthesis of (+)-nojirimycin and (+)-1-deoxynojirimycin. J Org Chem 1987, 52(15): 3337.
    • (1987) J Org Chem , vol.52 , Issue.15 , pp. 3337
    • Iida, H.1
  • 3
    • 0002096855 scopus 로고
    • 1,4-Bismethanesulfonates of the stereoisomeric butanetetraols and related compounds
    • Feit, P.W. 1,4-Bismethanesulfonates of the stereoisomeric butanetetraols and related compounds. J Med Chem 1964, 7: 14-7.
    • (1964) J Med Chem , vol.7 , pp. 14-17
    • Feit, P.W.1
  • 4
    • 0034957223 scopus 로고    scopus 로고
    • A novel approach to both the enantiomers of potent glycosidase inhibitor isofagomine via PET-promoted cyclization of 1-[benzyl(trimethylsilylmethyl)amino]-1,4,5-trideoxy-2,3-0-(1-methylethylidene) -threo-pent-4-ynitol
    • Pandey, G., Kapur, M. A novel approach to both the enantiomers of potent glycosidase inhibitor isofagomine via PET-promoted cyclization of 1-[benzyl(trimethylsilylmethyl)amino]-1,4,5-trideoxy-2,3-0-(1-methylethylidene) -threo-pent-4-ynitol. Synthesis (Stuttgart) 2001, (8): 1263-7.
    • (2001) Synthesis (Stuttgart) , vol.8 , pp. 1263-1267
    • Pandey, G.1    Kapur, M.2
  • 5
    • 0028035151 scopus 로고
    • Synthesis of isofagomine, a novel glycosidase inhibitor
    • Jespersen, T.M., Bols, M., Sierks, M.R., Skrydstrup, T. Synthesis of isofagomine, a novel glycosidase inhibitor. Tetrahedron 1994, 50(47): 13449-60.
    • (1994) Tetrahedron , vol.50 , Issue.47 , pp. 13449-13460
    • Jespersen, T.M.1    Bols, M.2    Sierks, M.R.3    Skrydstrup, T.4
  • 6
    • 76649101987 scopus 로고    scopus 로고
    • Lundgren, K, Kirk, O, Novo Nordisk A/S, Piperidines and pyrrolidines. EP 0749423, JP1997509947, JP 2008056688, WO 1995024391
    • Lundgren, K., Kirk, O. (Novo Nordisk A/S). Piperidines and pyrrolidines. EP 0749423, JP1997509947, JP 2008056688, WO 1995024391.
  • 7
    • 0034696490 scopus 로고    scopus 로고
    • Synthesis of (±)-isofagomine and its stereoisomers from arecoline
    • Hansen, S.U., Bols, M. Synthesis of (±)-isofagomine and its stereoisomers from arecoline. J Chem Soc Perkin Trans I 2000, (6): 911-5.
    • (2000) J Chem Soc Perkin Trans I , vol.6 , pp. 911-915
    • Hansen, S.U.1    Bols, M.2
  • 8
    • 0035098982 scopus 로고    scopus 로고
    • Iminosugars: Potential inhibitors of liver glycogen Phosphorylase
    • Jakobsen, P., Lundbeck, J.M., Kristiansen, M. et al. Iminosugars: Potential inhibitors of liver glycogen Phosphorylase. Bioorg Med Chem 2001, 9(3): 733-44.
    • (2001) Bioorg Med Chem , vol.9 , Issue.3 , pp. 733-744
    • Jakobsen, P.1    Lundbeck, J.M.2    Kristiansen, M.3
  • 9
    • 0035945791 scopus 로고    scopus 로고
    • Selective Fowler reductions: Asymmetric total syntheses of isofagomine and other 1-azasugars from methyl nicotinate
    • Zhao, G., Deo, U.C., Ganem, B. Selective Fowler reductions: Asymmetric total syntheses of isofagomine and other 1-azasugars from methyl nicotinate. Org Lett 2001, 3(2): 201-3.
    • (2001) Org Lett , vol.3 , Issue.2 , pp. 201-203
    • Zhao, G.1    Deo, U.C.2    Ganem, B.3
  • 10
    • 76649139364 scopus 로고    scopus 로고
    • Mugrace, B, Tretyakov, A, Zhu, X, Sheth, K.A, Rybczynski, P.J, Fuerst, D, Amicus Therapeutics, Inc, New method for preparing isofagomine and its derivatives. WO 2008144773
    • Mugrace, B., Tretyakov, A., Zhu, X., Sheth, K.A., Rybczynski, P.J., Fuerst, D. (Amicus Therapeutics, Inc.). New method for preparing isofagomine and its derivatives. WO 2008144773.
  • 11
    • 28044449165 scopus 로고    scopus 로고
    • Rational design and synthesis of highly potent beta-glucocerebrosidase inhibitors
    • Zhu, X., Sheth, A.K., Li, S., Chang, H.-H., Fan, J.-Q. Rational design and synthesis of highly potent beta-glucocerebrosidase inhibitors. Angew Chem lnt Ed 2005, 44(45): 7450-3.
    • (2005) Angew Chem lnt Ed , vol.44 , Issue.45 , pp. 7450-7453
    • Zhu, X.1    Sheth, A.K.2    Li, S.3    Chang, H.-H.4    Fan, J.-Q.5
  • 12
    • 0036433582 scopus 로고    scopus 로고
    • The synthesis of a carbohydrate-like dihydrooxazine and tetrahydrooxozine as putative inhibitors of glycoside hydrolases: A direct synthesis of isofagomine
    • Best, M.W., MacDonald, M.J., Skelton, W.B., Stick, R.V., Tilbrook,D.M.G., White, A.H. The synthesis of a carbohydrate-like dihydrooxazine and tetrahydrooxozine as putative inhibitors of glycoside hydrolases: A direct synthesis of isofagomine. Can J Chem 2002, 80: 857-65.
    • (2002) Can J Chem , vol.80 , pp. 857-865
    • Best, M.W.1    MacDonald, M.J.2    Skelton, W.B.3    Stick, R.V.4    Tilbrook, D.M.G.5    White, A.H.6
  • 13
    • 76649126108 scopus 로고    scopus 로고
    • Mugrage, B, Palling, D, Sheht, K.A, Rybczynski, P.J, Amicus Therapeutics, Inc, Tartrate salt of isofagomine and methods of use. EP1860101, JP 2007314540, WO 2007140212
    • Mugrage, B., Palling, D., Sheht, K.A., Rybczynski, P.J. (Amicus Therapeutics, Inc.). Tartrate salt of isofagomine and methods of use. EP1860101, JP 2007314540, WO 2007140212.
  • 14
    • 0035801470 scopus 로고    scopus 로고
    • Efficient synthesis of isofagomine and noeuromycin
    • Andersch, J., Bols, M. Efficient synthesis of isofagomine and noeuromycin. Chem Eur J 2001, 7(17): 3744.
    • (2001) Chem Eur J , vol.7 , Issue.17 , pp. 3744
    • Andersch, J.1    Bols, M.2
  • 15
    • 0037421035 scopus 로고    scopus 로고
    • Asymmetrized tris(hydroxymethyl)methane as precursor of iminosugars: Application to the synthesis of isofagomine
    • Guanti, G., Riva, R. Asymmetrized tris(hydroxymethyl)methane as precursor of iminosugars: Application to the synthesis of isofagomine. Tetrahedron Lett 2003, 44(2): 357-60.
    • (2003) Tetrahedron Lett , vol.44 , Issue.2 , pp. 357-360
    • Guanti, G.1    Riva, R.2
  • 16
    • 18844401427 scopus 로고    scopus 로고
    • Asymmetrized tris(hydroxymethyl)methane as a precursor of N- and O-containing 6-membered heterocycles through ring-closing metathesis
    • Banfi, L., Guanti, G., Paravidino, M., Riva, R. Asymmetrized tris(hydroxymethyl)methane as a precursor of N- and O-containing 6-membered heterocycles through ring-closing metathesis. Org Biomol Chem 2005, 3(9): 1729-37.
    • (2005) Org Biomol Chem , vol.3 , Issue.9 , pp. 1729-1737
    • Banfi, L.1    Guanti, G.2    Paravidino, M.3    Riva, R.4
  • 17
    • 20844458768 scopus 로고    scopus 로고
    • A new route to diverse 1-azasugars from N-Boc-5-hydroxy-3-piperidine as a common buiding block
    • Ouchi, H., Mihara, Y., Takahata, H. A new route to diverse 1-azasugars from N-Boc-5-hydroxy-3-piperidine as a common buiding block. J Org Chem 2005, 70: 5207-14.
    • (2005) J Org Chem , vol.70 , pp. 5207-5214
    • Ouchi, H.1    Mihara, Y.2    Takahata, H.3
  • 18
    • 4444354018 scopus 로고    scopus 로고
    • A short and concise synthesis of isofagomine, homoisofagomine, and 5-deoxyisofagomine
    • Ouchi, H., Mihara, Y., Watanabe, H., Takahata, H. A short and concise synthesis of isofagomine, homoisofagomine, and 5-deoxyisofagomine. Tetrahedron Lett 2004, 45(38): 7053-6.
    • (2004) Tetrahedron Lett , vol.45 , Issue.38 , pp. 7053-7056
    • Ouchi, H.1    Mihara, Y.2    Watanabe, H.3    Takahata, H.4
  • 19
    • 34547851826 scopus 로고    scopus 로고
    • Asymmetric synthesis of all stereoisomers of isofagomine using (2,3)-Wittig rearrangement
    • Mihara, Y., Ojima, H., Imahori, T., Yoshimura, Y., Ouchi, H., Takahata, H. Asymmetric synthesis of all stereoisomers of isofagomine using (2,3)-Wittig rearrangement. Heterocycles 2007, 72(1): 633-45.
    • (2007) Heterocycles , vol.72 , Issue.1 , pp. 633-645
    • Mihara, Y.1    Ojima, H.2    Imahori, T.3    Yoshimura, Y.4    Ouchi, H.5    Takahata, H.6
  • 20
    • 36849087357 scopus 로고    scopus 로고
    • Acceleration effect of allylic hydroxy group on ring-closing enyne metathesis of terminal alkynes: Scope and application to the synthesis of isofagomine
    • Imahori, T., Ojima, H., Tateyama, H., Mihara, Y., Takahata, H. Acceleration effect of allylic hydroxy group on ring-closing enyne metathesis of terminal alkynes: Scope and application to the synthesis of isofagomine. Tetrahedron Lett 2008, 49(2): 265-8.
    • (2008) Tetrahedron Lett , vol.49 , Issue.2 , pp. 265-268
    • Imahori, T.1    Ojima, H.2    Tateyama, H.3    Mihara, Y.4    Takahata, H.5
  • 21
    • 0013927537 scopus 로고
    • Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher's disease
    • Brady, R.O., Kanfer, J.N., Bradley, R.M., Shapiro, D. Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher's disease. J Clin Invest 1966, 45(7): 1112-5.
    • (1966) J Clin Invest , vol.45 , Issue.7 , pp. 1112-1115
    • Brady, R.O.1    Kanfer, J.N.2    Bradley, R.M.3    Shapiro, D.4
  • 22
    • 0027302227 scopus 로고
    • Gaucher disease. Enzymology, genetics, and treatment
    • Grabowski, G.A. Gaucher disease. Enzymology, genetics, and treatment. Adv Hum Genet 1993, 21: 377-441.
    • (1993) Adv Hum Genet , vol.21 , pp. 377-441
    • Grabowski, G.A.1
  • 23
    • 33745293617 scopus 로고    scopus 로고
    • Therapeutic strategies to ameliorate lysosomal storage disorders - A focus on Gaucher disease
    • Sawkar, A.R., D'Haeze, W., Kelly, J.W. Therapeutic strategies to ameliorate lysosomal storage disorders - A focus on Gaucher disease. Cell Mol Life Sci 2006, 63(10): 1179-92.
    • (2006) Cell Mol Life Sci , vol.63 , Issue.10 , pp. 1179-1192
    • Sawkar, A.R.1    D'Haeze, W.2    Kelly, J.W.3
  • 28
    • 33748801230 scopus 로고    scopus 로고
    • The iminosugar isofagomine increases the activity of N370S mutant acid beta-glucosidase in Gaucher fibroblasts by several mechanisms
    • Steet, R.A., Chung, S., Wustman, B., Powe, A., Do, H., Kornfeld, S.A. The iminosugar isofagomine increases the activity of N370S mutant acid beta-glucosidase in Gaucher fibroblasts by several mechanisms. Proc Natl Acad Sci USA 2006, 103(37): 13813-8.
    • (2006) Proc Natl Acad Sci USA , vol.103 , Issue.37 , pp. 13813-13818
    • Steet, R.A.1    Chung, S.2    Wustman, B.3    Powe, A.4    Do, H.5    Kornfeld, S.A.6
  • 29
    • 76649085115 scopus 로고    scopus 로고
    • Wustman, B.A., Khanna, R., Palling, D.J. et al. A new therapeutic approach to the treatment of Gaucher disease: Mechanisms of action of the pharmacological chaperone AT2101 and phase 1 trial results. 57th Annu Meet Am Soc Hum Genet (ASHG) (Oct 23-27, San Diego) 2007, Abst 2256/W.
    • Wustman, B.A., Khanna, R., Palling, D.J. et al. A new therapeutic approach to the treatment of Gaucher disease: Mechanisms of action of the pharmacological chaperone AT2101 and phase 1 trial results. 57th Annu Meet Am Soc Hum Genet (ASHG) (Oct 23-27, San Diego) 2007, Abst 2256/W.
  • 30
    • 76649084131 scopus 로고    scopus 로고
    • A new approach to the treatment of Gaucher's disease: Mechanism of enhancement of N370S GCase activity
    • March 21-25, Nashville, Abst 68
    • Do, H., Steet, R.A., Wustman, B. et al. A new approach to the treatment of Gaucher's disease: Mechanism of enhancement of N370S GCase activity. Annu Clin Genet Meet (March 21-25, Nashville) 2007, Abst 68.
    • (2007) Annu Clin Genet Meet
    • Do, H.1    Steet, R.A.2    Wustman, B.3
  • 31
    • 33947376087 scopus 로고    scopus 로고
    • Selective action of the iminosugar isofagomine, a pharmacological chaperone for mutant forms of acid-beta-glucosidase
    • Steet, R., Chung, S., Lee, W.S., Pine, C.W., Do, H., Kornfeld, S. Selective action of the iminosugar isofagomine, a pharmacological chaperone for mutant forms of acid-beta-glucosidase. Biochem Pharmacol 2007, 73(9): 1376-83.
    • (2007) Biochem Pharmacol , vol.73 , Issue.9 , pp. 1376-1383
    • Steet, R.1    Chung, S.2    Lee, W.S.3    Pine, C.W.4    Do, H.5    Kornfeld, S.6
  • 32
    • 76649090189 scopus 로고    scopus 로고
    • The pharmacological chaperone isofagomine increases L444P GCase levels in mice with a Gaucher disease-like phenotype
    • March 21-25, Nashville, Abst 67
    • Khanna, R., Soska, R., Lun, Y. et al. The pharmacological chaperone isofagomine increases L444P GCase levels in mice with a Gaucher disease-like phenotype. Annu Clin Genet Meet (March 21-25, Nashville) 2007, Abst 67.
    • (2007) Annu Clin Genet Meet
    • Khanna, R.1    Soska, R.2    Lun, Y.3
  • 33
    • 76649112640 scopus 로고    scopus 로고
    • Phase 1 studies of the safety, pharmacokinetics and pharmacodynamics of AT2101, a pharmacological chaperone or the treatment of Gaucher disease
    • March 21-25, Nashville, Abst 154
    • Palling, D.J., Ludwig, K., Casar, M. et al. Phase 1 studies of the safety, pharmacokinetics and pharmacodynamics of AT2101, a pharmacological chaperone or the treatment of Gaucher disease. Annu Clin Genet Meet (March 21-25, Nashville) 2007, Abst 154.
    • (2007) Annu Clin Genet Meet
    • Palling, D.J.1    Ludwig, K.2    Casar, M.3
  • 34
    • 76649092134 scopus 로고    scopus 로고
    • Pine, C.W., Ranes, B.E., Insinga, F. et al. The pharmacological chaperone AT2101 increases beta-glucocerebrosidase levels in macrophages and lymphoblasts derived from Gaucher patients. 57th Annu Meet Am Soc Hum Genet (ASHG) (Oct 23-27, San Diego) 2007, Abst 2254/W.
    • Pine, C.W., Ranes, B.E., Insinga, F. et al. The pharmacological chaperone AT2101 increases beta-glucocerebrosidase levels in macrophages and lymphoblasts derived from Gaucher patients. 57th Annu Meet Am Soc Hum Genet (ASHG) (Oct 23-27, San Diego) 2007, Abst 2254/W.
  • 35
    • 76649120761 scopus 로고    scopus 로고
    • A Gaucher disease ex vivo response study: The pharmacological chaperone AT2101 increases levels of glucocerebroside in patient-derived cells
    • June 16-19, Nice, Abst P1406
    • Pine, C.W., Ranes, B.E., Insinga, F. et al. A Gaucher disease ex vivo response study: The pharmacological chaperone AT2101 increases levels of glucocerebroside in patient-derived cells. 39th Eur Hum Genet Conf (June 16-19, Nice) 2007, Abst P1406.
    • (2007) 39th Eur Hum Genet Conf
    • Pine, C.W.1    Ranes, B.E.2    Insinga, F.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.