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763 related items for PubMed ID: 28040394

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

  • 2. Osteopenia in Gaucher disease develops early in life: response to imiglucerase enzyme therapy in children, adolescents and adults.
    Mistry PK, Weinreb NJ, Kaplan P, Cole JA, Gwosdow AR, Hangartner T.
    Blood Cells Mol Dis; 2011 Jan 15; 46(1):66-72. PubMed ID: 21112800
    [Abstract] [Full Text] [Related]

  • 3. Clinical-genetic characteristics and treatment outcomes of Turkish children with Gaucher disease type 1 and type 3: A sixteen year single-center experience.
    Gumus E, Karhan AN, Hizarcioglu-Gulsen H, Demir H, Ozen H, Saltik Temizel IN, Dokmeci Emre S, Yuce A.
    Eur J Med Genet; 2021 Nov 15; 64(11):104339. PubMed ID: 34500086
    [Abstract] [Full Text] [Related]

  • 4. 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]

  • 5. Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1.
    Andersson H, Kaplan P, Kacena K, Yee J.
    Pediatrics; 2008 Dec 15; 122(6):1182-90. PubMed ID: 19047232
    [Abstract] [Full Text] [Related]

  • 6. 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]

  • 7. 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]

  • 8. 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 15; 117(2):164-71. PubMed ID: 26043810
    [Abstract] [Full Text] [Related]

  • 9. Baseline characteristics of 32 patients with Gaucher disease who were treated with imiglucerase: South African data from the International Collaborative Gaucher Group (ICGG) Gaucher Registry.
    Sevittz H, Laher F, Varughese ST, Nel M, McMaster A, Jacobson BF.
    S Afr Med J; 2022 Feb 02; 112(1):13518. PubMed ID: 35140000
    [Abstract] [Full Text] [Related]

  • 10. Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment.
    Weinreb NJ, Goldblatt J, Villalobos J, Charrow J, Cole JA, Kerstenetzky M, vom Dahl S, Hollak C.
    J Inherit Metab Dis; 2013 May 02; 36(3):543-53. PubMed ID: 22976765
    [Abstract] [Full Text] [Related]

  • 11. [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 02; 44(9):653-6. PubMed ID: 17217655
    [Abstract] [Full Text] [Related]

  • 12. 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 02; 92(9):929-939. PubMed ID: 28569047
    [Abstract] [Full Text] [Related]

  • 13. 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 02; 94(8):647-51. PubMed ID: 15352589
    [Abstract] [Full Text] [Related]

  • 14. A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase.
    Pastores GM, Petakov M, Giraldo P, Rosenbaum H, Szer J, Deegan PB, Amato DJ, Mengel E, Tan ES, Chertkoff R, Brill-Almon E, Zimran A.
    Blood Cells Mol Dis; 2014 Dec 02; 53(4):253-60. PubMed ID: 24950666
    [Abstract] [Full Text] [Related]

  • 15. 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]

  • 16. Neuronopathic Gaucher disease presenting with microcytic hypochromic anemia.
    Kim EA, Lim YT, Hah JO, Sohn YB, Kim YK, Choi JH, Kim SY, Jang KM, Ahn J, Lee JM.
    Int J Hematol; 2019 Mar 13; 109(3):361-365. PubMed ID: 30456712
    [Abstract] [Full Text] [Related]

  • 17. Management of Gaucher disease: enzyme replacement therapy.
    Zimran A, Elstein D.
    Pediatr Endocrinol Rev; 2014 Sep 13; 12 Suppl 1():82-7. PubMed ID: 25345089
    [Abstract] [Full Text] [Related]

  • 18. 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]

  • 19. Gaucher disease: clinical phenotypes and refining GBA mutational spectrum in Thai patients.
    Phetthong T, Tim-Aroon T, Khongkraparn A, Noojarern S, Kuptanon C, Wichajarn K, Sathienkijkanchai A, Suphapeetiporn K, Charoenkwan P, Tantiworawit A, Noentong N, Wattanasirichaigoon D.
    Orphanet J Rare Dis; 2021 Dec 20; 16(1):519. PubMed ID: 34930372
    [Abstract] [Full Text] [Related]

  • 20. 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 20; 158(4):528-38. PubMed ID: 22640238
    [Abstract] [Full Text] [Related]

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