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Journal Abstract Search

196 related items for PubMed ID: 21978771

  • 1. Gaucher disease due to saposin C deficiency, previously described as non-neuronopathic form--no positive effects after 2-years of miglustat therapy.
    Tylki-Szymańska A, Groener JE, Kamiński ML, Ługowska A, Jurkiewicz E, Czartoryska B.
    Mol Genet Metab; 2011 Dec; 104(4):627-30. PubMed ID: 21978771
    [Abstract] [Full Text] [Related]

  • 2. Non-neuronopathic Gaucher disease due to saposin C deficiency.
    Tylki-Szymańska A, Czartoryska B, Vanier MT, Poorthuis BJ, Groener JA, Ługowska A, Millat G, Vaccaro AM, Jurkiewicz E.
    Clin Genet; 2007 Dec; 72(6):538-42. PubMed ID: 17919309
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  • 3. An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment.
    Pastores GM, Barnett NL, Kolodny EH.
    Clin Ther; 2005 Aug; 27(8):1215-27. PubMed ID: 16199246
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  • 4. Neurologic improvement in a type 3 Gaucher disease patient treated with imiglucerase/miglustat combination.
    Capablo JL, Franco R, de Cabezón AS, Alfonso P, Pocovi M, Giraldo P.
    Epilepsia; 2007 Jul; 48(7):1406-8. PubMed ID: 17433057
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  • 6. Treatment of Gaucher's disease with OGT 918.
    Mistry PK.
    Lancet; 2000 Aug 19; 356(9230):676-7. PubMed ID: 10968454
    [No Abstract] [Full Text] [Related]

  • 7. Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies.
    Pastores GM, Elstein D, Hrebícek M, Zimran A.
    Clin Ther; 2007 Aug 19; 29(8):1645-54. PubMed ID: 17919546
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  • 9. Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease.
    Moyses C.
    Philos Trans R Soc Lond B Biol Sci; 2003 May 29; 358(1433):955-60. PubMed ID: 12803929
    [Abstract] [Full Text] [Related]

  • 10. [Organization of Gaucher disease management in France].
    Stirnemann J, de Villemeur TB, Belmatoug N.
    Rev Med Interne; 2007 Oct 29; 28 Suppl 2():S198-201. PubMed ID: 18228688
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  • 11. Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report.
    Giuffrida G, Lombardo R, Di Francesco E, Parrinello L, Di Raimondo F, Fiumara A.
    J Med Case Rep; 2016 Nov 08; 10(1):315. PubMed ID: 27821156
    [Abstract] [Full Text] [Related]

  • 12. Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme.
    Hollak CE, Hughes D, van Schaik IN, Schwierin B, Bembi B.
    Pharmacoepidemiol Drug Saf; 2009 Sep 08; 18(9):770-7. PubMed ID: 19507165
    [Abstract] [Full Text] [Related]

  • 13. Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients.
    Smid BE, Ferraz MJ, Verhoek M, Mirzaian M, Wisse P, Overkleeft HS, Hollak CE, Aerts JM.
    Orphanet J Rare Dis; 2016 Mar 24; 11():28. PubMed ID: 27008851
    [Abstract] [Full Text] [Related]

  • 14. Substrate reduction therapy with miglustat for type 1 Gaucher disease: a retrospective analysis from a single institution.
    Machaczka M, Hast R, Dahlman I, Lerner R, Klimkowska M, Engvall M, Hägglund H.
    Ups J Med Sci; 2012 Mar 24; 117(1):28-34. PubMed ID: 22247978
    [Abstract] [Full Text] [Related]

  • 15. Results from a 9-year Intensive Safety Surveillance Scheme (IS(3) ) in miglustat (Zavesca(®) )-treated patients.
    Brand M, Muller A, Alsop J, van Schaik IN, Bembi B, Hughes D.
    Pharmacoepidemiol Drug Saf; 2015 Mar 24; 24(3):329-33. PubMed ID: 25656910
    [Abstract] [Full Text] [Related]

  • 16. Patients with Gaucher type 1: Switching from imiglucerase to miglustat therapy.
    Canda E, Kose M, Kagnici M, Ucar SK, Sozmen EY, Coker M.
    Blood Cells Mol Dis; 2018 Feb 24; 68():180-184. PubMed ID: 28111116
    [No Abstract] [Full Text] [Related]

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  • 19. Misdiagnosis of Niemann-Pick disease type C as Gaucher disease.
    Lo SM, McNamara J, Seashore MR, Mistry PK.
    J Inherit Metab Dis; 2010 Dec 24; 33 Suppl 3(0 3):S429-33. PubMed ID: 20882348
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