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Journal Abstract Search

226 related items for PubMed ID: 8392397

  • 21. Individualization of long-term enzyme replacement therapy for Gaucher disease.
    Andersson HC, Charrow J, Kaplan P, Mistry P, Pastores GM, Prakash-Cheng A, Rosenbloom BE, Scott CR, Wappner RS, Weinreb NJ, International Collaborative Gaucher Group U.S. Regional Coordinators.
    Genet Med; 2005 Feb; 7(2):105-10. PubMed ID: 15714077
    [Abstract] [Full Text] [Related]

  • 22. Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease.
    Barton NW, Brady RO, Dambrosia JM, Di Bisceglie AM, Doppelt SH, Hill SC, Mankin HJ, Murray GJ, Parker RI, Argoff CE.
    N Engl J Med; 1991 May 23; 324(21):1464-70. PubMed ID: 2023606
    [Abstract] [Full Text] [Related]

  • 23. Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial.
    Lukina E, Watman N, Dragosky M, Lau H, Avila Arreguin E, Rosenbaum H, Zimran A, Foster MC, Gaemers SJM, Peterschmitt MJ.
    Am J Hematol; 2019 Jan 23; 94(1):29-38. PubMed ID: 30264864
    [Abstract] [Full Text] [Related]

  • 24. Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.
    Mistry PK, Batista JL, Andersson HC, Balwani M, Burrow TA, Charrow J, Kaplan P, Khan A, Kishnani PS, Kolodny EH, Rosenbloom B, Scott CR, Weinreb N.
    Am J Hematol; 2017 Sep 23; 92(9):929-939. PubMed ID: 28569047
    [Abstract] [Full Text] [Related]

  • 25. Differential effects of enzyme supplementation therapy on manifestations of type 1 Gaucher disease.
    Hollak CE, Corssmit EP, Aerts JM, Endert E, Sauerwein HP, Romijn JA, van Oers MH.
    Am J Med; 1997 Sep 23; 103(3):185-91. PubMed ID: 9316550
    [Abstract] [Full Text] [Related]

  • 26. Enzyme therapy in Gaucher disease type 1: effect of neutralizing antibodies to acid beta-glucosidase.
    Ponce E, Moskovitz J, Grabowski G.
    Blood; 1997 Jul 01; 90(1):43-8. PubMed ID: 9207436
    [Abstract] [Full Text] [Related]

  • 27. Enzyme replacement therapy for Gaucher disease.
    Beutler E, Kay A, Saven A, Garver P, Thurston D, Dawson A, Rosenbloom B.
    Blood; 1991 Sep 01; 78(5):1183-9. PubMed ID: 1878585
    [Abstract] [Full Text] [Related]

  • 28. A phase 2 multi-center, open-label, switch-over trial to evaluate the safety and efficacy of Abcertin® in patients with type 1 Gaucher disease.
    Choi JH, Lee BH, Ko JM, Sohn YB, Lee JS, Kim GH, Heo SH, Park JY, Kim YM, Kim JH, Yoo HW.
    J Korean Med Sci; 2015 Apr 01; 30(4):378-84. PubMed ID: 25829804
    [Abstract] [Full Text] [Related]

  • 29. [Type I Gaucher's disease: clinical, evolutive and therapeutic features in 8 cases].
    Giraldo P, Pérez-Calvo J, Cortés T, Civeira F, Rubio-Félix D.
    Sangre (Barc); 1994 Feb 01; 39(1):3-7. PubMed ID: 8197516
    [Abstract] [Full Text] [Related]

  • 30. The clinical and demographic characteristics of nonneuronopathic Gaucher disease in 887 children at diagnosis.
    Kaplan P, Andersson HC, Kacena KA, Yee JD.
    Arch Pediatr Adolesc Med; 2006 Jun 01; 160(6):603-8. PubMed ID: 16754822
    [Abstract] [Full Text] [Related]

  • 31. A randomized trial comparing the efficacy and safety of imiglucerase (Cerezyme) infusions every 4 weeks versus every 2 weeks in the maintenance therapy of adult patients with Gaucher disease type 1.
    Kishnani PS, DiRocco M, Kaplan P, Mehta A, Pastores GM, Smith SE, Puga AC, Lemay RM, Weinreb NJ.
    Mol Genet Metab; 2009 Apr 01; 96(4):164-70. PubMed ID: 19195916
    [Abstract] [Full Text] [Related]

  • 32. Enzyme replacement therapy for Gaucher disease: skeletal responses to macrophage-targeted glucocerebrosidase.
    Rosenthal DI, Doppelt SH, Mankin HJ, Dambrosia JM, Xavier RJ, McKusick KA, Rosen BR, Baker J, Niklason LT, Hill SC.
    Pediatrics; 1995 Oct 01; 96(4 Pt 1):629-37. PubMed ID: 7567322
    [Abstract] [Full Text] [Related]

  • 33. Imiglucerase low-dose therapy for paediatric Gaucher disease--a long-term cohort study.
    Heitner R, Arndt S, Levin JB.
    S Afr Med J; 2004 Aug 01; 94(8):647-51. PubMed ID: 15352589
    [Abstract] [Full Text] [Related]

  • 34. Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naïve patients with Gaucher disease.
    Zimran A, Durán G, Giraldo P, Rosenbaum H, Giona F, Petakov M, Terreros Muñoz E, Solorio-Meza SE, Cooper PA, Varughese S, Alon S, Chertkoff R.
    Blood Cells Mol Dis; 2019 Sep 01; 78():14-21. PubMed ID: 27499018
    [Abstract] [Full Text] [Related]

  • 35. Home treatment with intravenous enzyme replacement therapy for Gaucher disease: an international collaborative study of 33 patients.
    Zimran A, Hollak CE, Abrahamov A, van Oers MH, Kelly M, Beutler E.
    Blood; 1993 Aug 15; 82(4):1107-9. PubMed ID: 8353277
    [Abstract] [Full Text] [Related]

  • 36. Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 study.
    Gonzalez DE, Turkia HB, Lukina EA, Kisinovsky I, Dridi MF, Elstein D, Zahrieh D, Crombez E, Bhirangi K, Barton NW, Zimran A.
    Am J Hematol; 2013 Mar 15; 88(3):166-71. PubMed ID: 23386328
    [Abstract] [Full Text] [Related]

  • 37. Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry.
    El-Beshlawy A, Tylki-Szymanska A, Vellodi A, Belmatoug N, Grabowski GA, Kolodny EH, Batista JL, Cox GF, Mistry PK.
    Mol Genet Metab; 2017 Mar 15; 120(1-2):47-56. PubMed ID: 28040394
    [Abstract] [Full Text] [Related]

  • 38. [Gaucher disease type 1--therapeutic results of enzyme substitution].
    Steensberg J, Nielsen KG, Brandt NJ.
    Ugeskr Laeger; 1998 Jun 22; 160(26):3929-30. PubMed ID: 9656836
    [Abstract] [Full Text] [Related]

  • 39. The clinical course of untreated Gaucher disease in 22 patients over 10 years: hematological and skeletal manifestations.
    Piran S, Roberts A, Patterson MA, Amato D.
    Blood Cells Mol Dis; 2009 Jun 22; 43(3):289-93. PubMed ID: 19793665
    [Abstract] [Full Text] [Related]

  • 40. Mutation analysis of the acid beta-glucosidase gene in a patient with type 3 Gaucher disease and neutralizing antibody to alglucerase.
    Germain DP, Kaneski CR, Brady RO.
    Mutat Res; 2001 Nov 01; 483(1-2):89-94. PubMed ID: 11600137
    [Abstract] [Full Text] [Related]

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