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Volumn 32, Issue SUPPL. 1, 2009, Pages

Miglustat therapy in juvenile Sandhoff disease

Author keywords

[No Author keywords available]

Indexed keywords

1 DEOXYNOJIRIMYCIN; BETA N ACETYLHEXOSAMINIDASE BETA CHAIN; CERAMIDE GLUCOSYLTRANSFERASE; DRUG DERIVATIVE; ENZYME INHIBITOR; GLUCOSYLTRANSFERASE; HEXB PROTEIN, HUMAN; MIGLUSTAT;

EID: 84880990833     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-009-1224-7     Document Type: Article
Times cited : (19)

References (15)
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    • 57749103607 scopus 로고    scopus 로고
    • Substrate reduction therapy in Sandhoff disease: Evidence for improvement in nervous function in patients treated with miglustat
    • Lachmann RH, Wright N, Parker A et al. (2006) Substrate reduction therapy in Sandhoff disease: evidence for improvement in nervous function in patients treated with miglustat. J Inherit Metab Dis 29(Suppl 1):130
    • (2006) J Inherit Metab Dis , vol.29 , Issue.SUPPL. 1 , pp. 130
    • Lachmann, R.H.1    Wright, N.2    Parker, A.3
  • 6
    • 33750944986 scopus 로고    scopus 로고
    • 2 gangliosidosis: 21 new cases and literature review of 134 previously reported
    • 2 gangliosidosis: 21 new cases and literature review of 134 previously reported. Pediatrics 118:e1550-1562
    • (2006) Pediatrics , vol.118
    • Maegawa, G.H.1    Stockley, T.2    Tropak, M.3
  • 11
    • 0028176432 scopus 로고
    • N-Butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis
    • Platt FM, Neises GR, Dwek RA, Butters TD (1994) N-Butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis. J Biol Chem 269:8362-8365
    • (1994) J Biol Chem , vol.269 , pp. 8362-8365
    • Platt, F.M.1    Neises, G.R.2    Dwek, R.A.3    Butters, T.D.4
  • 13
    • 0019984977 scopus 로고
    • Thiamin monophosphate in the CSF of patients with amyotrophic lateral sclerosis
    • Poloni M, Patrini C, Rocchelli B, Rindi G (1982) Thiamin monophosphate in the CSF of patients with amyotrophic lateral sclerosis. Arch Neurol 39:507-509 (Pubitemid 12089652)
    • (1982) Archives of Neurology , vol.39 , Issue.8 , pp. 507-509
    • Poloni, M.1    Patrini, C.2    Rocchelli, B.3    Rindi, G.4
  • 15
    • 33947314170 scopus 로고    scopus 로고
    • The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat
    • DOI 10.1080/00498250601094543, PII 773288053
    • Treiber A, Morand O, Clozel M (2007) The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat. Xenobiotica 37:298-314 (Pubitemid 46439676)
    • (2007) Xenobiotica , vol.37 , Issue.3 , pp. 298-314
    • Treiber, A.1    Morand, O.2    Clozel, M.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.