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Journal Abstract Search

152 related items for PubMed ID: 29471591

  • 1. Improvement of life quality measured by Lansky Score after enzymatic replacement therapy in children with Gaucher disease type 1.
    Cerón-Rodríguez M, Barajas-Colón E, Ramírez-Devars L, Gutiérrez-Camacho C, Salgado-Loza JL.
    Mol Genet Genomic Med; 2018 Jan; 6(1):27-34. PubMed ID: 29471591
    [Abstract] [Full Text] [Related]

  • 2. [A retrospective study on enzyme replacement therapy in patients with Gaucher disease].
    Duan YL, Zhang YH, Zang Y, Shi HP, Zhang WM, Hu YM.
    Zhonghua Er Ke Za Zhi; 2006 Sep; 44(9):653-6. PubMed ID: 17217655
    [Abstract] [Full Text] [Related]

  • 3. 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]

  • 4. 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 27; 120(1-2):47-56. PubMed ID: 28040394
    [Abstract] [Full Text] [Related]

  • 5. Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis.
    Mistry PK, Deegan P, Vellodi A, Cole JA, Yeh M, Weinreb NJ.
    Br J Haematol; 2009 Nov 27; 147(4):561-70. PubMed ID: 19732054
    [Abstract] [Full Text] [Related]

  • 6. Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase.
    Smith L, Rhead W, Charrow J, Shankar SP, Bavdekar A, Longo N, Mardach R, Harmatz P, Hangartner T, Lee HM, Crombez E, Pastores GM.
    Mol Genet Metab; 2016 Feb 27; 117(2):164-71. PubMed ID: 26043810
    [Abstract] [Full Text] [Related]

  • 7. 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 27; 92(9):929-939. PubMed ID: 28569047
    [Abstract] [Full Text] [Related]

  • 8. Booster-effect with velaglucerase alfa in patients with Gaucher disease switched from long-term imiglucerase therapy: early Access Program results from Jerusalem.
    Elstein D, Altarescu G, Maayan H, Phillips M, Abrahamov A, Hadas-Halpern I, Tiomkin M, Zimran A.
    Blood Cells Mol Dis; 2012 Jan 15; 48(1):45-50. PubMed ID: 22047948
    [Abstract] [Full Text] [Related]

  • 9. Dynamic changes of lipid profile in Romanian patients with Gaucher disease type 1 under enzyme replacement therapy: a prospective study.
    Zimmermann A, Grigorescu-Sido P, Rossmann H, Lackner KJ, Drugan C, Al Khzouz C, Bucerzan S, Naşcu I, Zimmermann T, Leucuţa D, Weber MM.
    J Inherit Metab Dis; 2013 May 15; 36(3):555-63. PubMed ID: 22976766
    [Abstract] [Full Text] [Related]

  • 10. Focal splenic lesions in type I Gaucher disease are associated with poor platelet and splenic response to macrophage-targeted enzyme replacement therapy.
    Stein P, Malhotra A, Haims A, Pastores GM, Mistry PK.
    J Inherit Metab Dis; 2010 Dec 15; 33(6):769-74. PubMed ID: 20683668
    [Abstract] [Full Text] [Related]

  • 11. Velaglucerase alfa for the management of type 1 Gaucher disease.
    Morris JL.
    Clin Ther; 2012 Feb 15; 34(2):259-71. PubMed ID: 22264444
    [Abstract] [Full Text] [Related]

  • 12. Enzyme replacement therapy with imiglucerase in a Taiwanese child with type 1 Gaucher disease.
    Lin HY, Lin SP, Chuang CK, Wraith JE.
    J Chin Med Assoc; 2006 May 15; 69(5):228-32. PubMed ID: 16835986
    [Abstract] [Full Text] [Related]

  • 13. Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase.
    Zimran A, Pastores GM, Tylki-Szymanska A, Hughes DA, Elstein D, Mardach R, Eng C, Smith L, Heisel-Kurth M, Charrow J, Harmatz P, Fernhoff P, Rhead W, Longo N, Giraldo P, Ruiz JA, Zahrieh D, Crombez E, Grabowski GA.
    Am J Hematol; 2013 Mar 15; 88(3):172-8. PubMed ID: 23339116
    [Abstract] [Full Text] [Related]

  • 14. Effect of enzyme replacement therapy with imiglucerase on BMD in type 1 Gaucher disease.
    Wenstrup RJ, Kacena KA, Kaplan P, Pastores GM, Prakash-Cheng A, Zimran A, Hangartner TN.
    J Bone Miner Res; 2007 Jan 15; 22(1):119-26. PubMed ID: 17032149
    [Abstract] [Full Text] [Related]

  • 15. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment.
    Weinreb NJ, Camelo JS, Charrow J, McClain MR, Mistry P, Belmatoug N, International Collaborative Gaucher Group (ICGG) Gaucher Registry (NCT00358943) investigators.
    Mol Genet Metab; 2021 Feb 15; 132(2):100-111. PubMed ID: 33485799
    [Abstract] [Full Text] [Related]

  • 16. Clinical consequences of interrupting enzyme replacement therapy in children with type 1 Gaucher disease.
    Drelichman G, Ponce E, Basack N, Freigeiro D, Aversa L, Graciela E, Kohan R.
    J Pediatr; 2007 Aug 15; 151(2):197-201. PubMed ID: 17643778
    [Abstract] [Full Text] [Related]

  • 17. Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1.
    Stirnemann J, Rose C, Serratrice C, Dalbies F, Lidove O, Masseau A, Pers YM, Baron C, Belmatoug N.
    Orphanet J Rare Dis; 2015 May 13; 10():62. PubMed ID: 25968608
    [Abstract] [Full Text] [Related]

  • 18. 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 13; 88(3):166-71. PubMed ID: 23386328
    [Abstract] [Full Text] [Related]

  • 19. Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years.
    Hollak CE, Belmatoug N, Cole JA, Vom Dahl S, Deegan PB, Goldblatt J, Rosenbloom B, van Dussen L, Tylki-Szymańska A, Weinreb NJ, Zimran A, Cappellini MD.
    Br J Haematol; 2012 Aug 13; 158(4):528-38. PubMed ID: 22640238
    [Abstract] [Full Text] [Related]

  • 20. Long-term effectiveness of enzyme replacement therapy in children with Gaucher disease: results from the NCS-LSD cohort study.
    Anderson LJ, Henley W, Wyatt KM, Nikolaou V, Waldek S, Hughes DA, Pastores GM, Logan S.
    J Inherit Metab Dis; 2014 Nov 13; 37(6):961-8. PubMed ID: 24638276
    [Abstract] [Full Text] [Related]

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