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Journal Abstract Search

572 related items for PubMed ID: 28103924

  • 1. Combination therapy in a patient with chronic neuronopathic Gaucher disease: a case report.
    Ceravolo F, Grisolia M, Sestito S, Falvo F, Moricca MT, Concolino D.
    J Med Case Rep; 2017 Jan 20; 11(1):19. PubMed ID: 28103924
    [Abstract] [Full Text] [Related]

  • 2. Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports.
    Amato D, Patterson MA.
    J Med Case Rep; 2018 Jan 27; 12(1):19. PubMed ID: 29373994
    [Abstract] [Full Text] [Related]

  • 3. 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]

  • 4. Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series.
    Serratrice C, Swiader L, Serratrice J.
    J Med Case Rep; 2015 Jun 23; 9():146. PubMed ID: 26100396
    [Abstract] [Full Text] [Related]

  • 5. Enzyme replacement and substrate reduction therapy for Gaucher disease.
    Shemesh E, Deroma L, Bembi B, Deegan P, Hollak C, Weinreb NJ, Cox TM.
    Cochrane Database Syst Rev; 2015 Mar 27; 2015(3):CD010324. PubMed ID: 25812601
    [Abstract] [Full Text] [Related]

  • 6. Substrate reduction therapy of glycosphingolipid storage disorders.
    Aerts JM, Hollak CE, Boot RG, Groener JE, Maas M.
    J Inherit Metab Dis; 2006 Mar 27; 29(2-3):449-56. PubMed ID: 16763917
    [Abstract] [Full Text] [Related]

  • 7. 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]

  • 8. 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 24; 27(8):1215-27. PubMed ID: 16199246
    [Abstract] [Full Text] [Related]

  • 9. Value of plasma chitotriosidase to assess non-neuronopathic Gaucher disease severity and progression in the era of enzyme replacement therapy.
    van Dussen L, Hendriks EJ, Groener JE, Boot RG, Hollak CE, Aerts JM.
    J Inherit Metab Dis; 2014 Nov 24; 37(6):991-1001. PubMed ID: 24831585
    [Abstract] [Full Text] [Related]

  • 10. 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]

  • 11. 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 29; 48(7):1406-8. PubMed ID: 17433057
    [Abstract] [Full Text] [Related]

  • 12. Effective treatment of an elderly patient with Gaucher's disease and Parkinsonism: a case report of 24 months' oral substrate reduction therapy with miglustat.
    Hughes DA, Ginsberg L, Baker R, Goodwin S, Milligan A, Richfield L, Mehta AB.
    Parkinsonism Relat Disord; 2007 Aug 29; 13(6):365-8. PubMed ID: 17049454
    [Abstract] [Full Text] [Related]

  • 13. Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.
    Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P.
    Am J Hematol; 2005 Nov 29; 80(3):223-9. PubMed ID: 16247743
    [Abstract] [Full Text] [Related]

  • 14. Substrate reduction therapy.
    Platt FM, Jeyakumar M.
    Acta Paediatr; 2008 Apr 29; 97(457):88-93. PubMed ID: 18339196
    [Abstract] [Full Text] [Related]

  • 15. 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 29; 104(4):627-30. PubMed ID: 21978771
    [Abstract] [Full Text] [Related]

  • 16. Thirty-year clinical outcomes after haematopoietic stem cell transplantation in neuronopathic Gaucher disease.
    Donald A, Björkvall CK, Vellodi A, GAUCHERITE Consortium, Cox TM, Hughes D, Jones SA, Wynn R, Machaczka M.
    Orphanet J Rare Dis; 2022 Jun 18; 17(1):234. PubMed ID: 35717194
    [Abstract] [Full Text] [Related]

  • 17. 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 18; 64(5):514-22. PubMed ID: 19067373
    [Abstract] [Full Text] [Related]

  • 18. 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 18; 18(9):770-7. PubMed ID: 19507165
    [Abstract] [Full Text] [Related]

  • 19. Pharmacological treatment of pediatric Gaucher disease.
    Gupta P, Pastores G.
    Expert Rev Clin Pharmacol; 2018 Dec 18; 11(12):1183-1194. PubMed ID: 30444430
    [Abstract] [Full Text] [Related]

  • 20. Systemic enzyme delivery by blood-brain barrier-penetrating SapC-DOPS nanovesicles for treatment of neuronopathic Gaucher disease.
    Sun Y, Liou B, Chu Z, Fannin V, Blackwood R, Peng Y, Grabowski GA, Davis HW, Qi X.
    EBioMedicine; 2020 May 18; 55():102735. PubMed ID: 32279952
    [Abstract] [Full Text] [Related]

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