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Journal Abstract Search

201 related items for PubMed ID: 19346952

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  • 3. Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project.
    Giraldo P, Alfonso P, Atutxa K, Fernández-Galán MA, Barez A, Franco R, Alonso D, Martin A, Latre P, Pocovi M.
    Haematologica; 2009 Dec; 94(12):1771-5. PubMed ID: 19608672
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  • 4. Efficacy of miglustat in Niemann-Pick C disease: a single centre experience.
    Ginocchio VM, D'Amico A, Bertini E, Ceravolo F, Dardis A, Verrigni D, Bembi B, Dionisi-Vici C, Deodato F.
    Mol Genet Metab; 2013 Nov; 110(3):329-35. PubMed ID: 23973268
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  • 6. Randomized, controlled trial of miglustat in Gaucher's disease type 3.
    Schiffmann R, Fitzgibbon EJ, Harris C, DeVile C, Davies EH, Abel L, van Schaik IN, Benko W, Timmons M, Ries M, Vellodi A.
    Ann Neurol; 2008 Nov; 64(5):514-22. PubMed ID: 19067373
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  • 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; 29(8):1645-54. PubMed ID: 17919546
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  • 9. Efficacy and safety of miglustat in the treatment of GM2 gangliosidosis: A systematic review.
    Mansouri V, Tavasoli AR, Khodarahmi M, Dakkali MS, Daneshfar S, Ashrafi MR, Heidari M, Hosseinpour S, Sharifianjazi F, Bemanalizadeh M.
    Eur J Neurol; 2023 Sep; 30(9):2919-2945. PubMed ID: 37209042
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  • 11. Miglustat in adult and juvenile patients with Niemann-Pick disease type C: long-term data from a clinical trial.
    Wraith JE, Vecchio D, Jacklin E, Abel L, Chadha-Boreham H, Luzy C, Giorgino R, Patterson MC.
    Mol Genet Metab; 2010 Apr; 99(4):351-7. PubMed ID: 20045366
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  • 12. Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.
    Elstein D, Hollak C, Aerts JM, van Weely S, Maas M, Cox TM, Lachmann RH, Hrebicek M, Platt FM, Butters TD, Dwek RA, Zimran A.
    J Inherit Metab Dis; 2004 Apr; 27(6):757-66. PubMed ID: 15505381
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  • 13. Normalisation of brain spectroscopy findings in Niemann-Pick disease type C patients treated with miglustat.
    Sedel F, Chabrol B, Audoin B, Kaphan E, Tranchant C, Burzykowski T, Tourbah A, Vanier MT, Galanaud D.
    J Neurol; 2016 May; 263(5):927-936. PubMed ID: 26984608
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  • 14. Substrate reduction therapy in the infantile form of Tay-Sachs disease.
    Bembi B, Marchetti F, Guerci VI, Ciana G, Addobbati R, Grasso D, Barone R, Cariati R, Fernandez-Guillen L, Butters T, Pittis MG.
    Neurology; 2006 Jan 24; 66(2):278-80. PubMed ID: 16434676
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  • 15. Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C.
    Héron B, Valayannopoulos V, Baruteau J, Chabrol B, Ogier H, Latour P, Dobbelaere D, Eyer D, Labarthe F, Maurey H, Cuisset JM, de Villemeur TB, Sedel F, Vanier MT.
    Orphanet J Rare Dis; 2012 Jun 07; 7():36. PubMed ID: 22676771
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  • 16. Pharmacokinetics, safety and tolerability of miglustat in the treatment of pediatric patients with GM2 gangliosidosis.
    Maegawa GH, van Giersbergen PL, Yang S, Banwell B, Morgan CP, Dingemanse J, Tifft CJ, Clarke JT.
    Mol Genet Metab; 2009 Aug 07; 97(4):284-91. PubMed ID: 19447653
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  • 17. Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect.
    Nadjar Y, Hütter-Moncada AL, Latour P, Ayrignac X, Kaphan E, Tranchant C, Cintas P, Degardin A, Goizet C, Laurencin C, Martzolff L, Tilikete C, Anheim M, Audoin B, Deramecourt V, De Gaillarbois TD, Roze E, Lamari F, Vanier MT, Héron B.
    Orphanet J Rare Dis; 2018 Oct 01; 13(1):175. PubMed ID: 30285904
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  • 18. Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.
    Elstein D, Dweck A, Attias D, Hadas-Halpern I, Zevin S, Altarescu G, Aerts JF, van Weely S, Zimran A.
    Blood; 2007 Oct 01; 110(7):2296-301. PubMed ID: 17609429
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  • 19. A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat.
    Remenova T, Morand O, Amato D, Chadha-Boreham H, Tsurutani S, Marquardt T.
    Orphanet J Rare Dis; 2015 Jun 19; 10():81. PubMed ID: 26084276
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