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Items: 1 to 20 of 149

1.

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;158(4):528-38. doi: 10.1111/j.1365-2141.2012.09175.x. Epub 2012 May 29.

PMID:
22640238
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. Chinese.

PMID:
17217655
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;(3):CD010324. doi: 10.1002/14651858.CD010324.pub2. Review.

PMID:
25812601
4.

Velaglucerase alfa for the management of type 1 Gaucher disease.

Morris JL.

Clin Ther. 2012 Feb;34(2):259-71. doi: 10.1016/j.clinthera.2011.12.017. Epub 2012 Jan 20. Review.

PMID:
22264444
5.

Outcome of enzyme replacement therapy in patients with Gaucher disease type I. The Romanian experience.

Grigorescu Sido P, Drugan C, Cret V, Al-Kzouz C, Denes C, Coldea C, Zimmermann A.

J Inherit Metab Dis. 2007 Oct;30(5):783-9. Epub 2007 Aug 20.

PMID:
17703370
6.

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. doi: 10.1016/j.bcmd.2010.10.011. Epub 2010 Nov 26.

7.

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;36(3):543-53. doi: 10.1007/s10545-012-9528-4. Epub 2012 Sep 14. Erratum in: J Inherit Metab Dis. 2014 Jan;37(1):147.

8.

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 Jan - Feb;120(1-2):47-56. doi: 10.1016/j.ymgme.2016.12.001. Epub 2016 Dec 6.

9.

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase.

Weinreb N, Taylor J, Cox T, Yee J, vom Dahl S.

Am J Hematol. 2008 Dec;83(12):890-5. doi: 10.1002/ajh.21280.

10.

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;22(1):119-26.

11.

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;33(6):769-74. doi: 10.1007/s10545-010-9175-6. Epub 2010 Aug 4.

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;92(9):929-939. doi: 10.1002/ajh.24801. Epub 2017 Jul 7.

PMID:
28569047
13.

Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease.

Ben Turkia H, Gonzalez DE, Barton NW, Zimran A, Kabra M, Lukina EA, Giraldo P, Kisinovsky I, Bavdekar A, Ben Dridi MF, Gupta N, Kishnani PS, Sureshkumar EK, Wang N, Crombez E, Bhirangi K, Mehta A.

Am J Hematol. 2013 Mar;88(3):179-84. doi: 10.1002/ajh.23382. Epub 2013 Feb 9. Erratum in: Am J Hematol. 2013 Jul;88(7):632.

14.

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;147(4):561-70. doi: 10.1111/j.1365-2141.2009.07872.x. Epub 2009 Sep 3.

15.

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;88(3):166-71. doi: 10.1002/ajh.23381. Epub 2013 Feb 6.

16.

Enzyme replacement therapy with imiglucerase in Taiwanese patients with type I Gaucher disease.

Hsu CC, Chien YH, Lai MY, Hwu WL.

J Formos Med Assoc. 2002 Sep;101(9):627-31.

PMID:
12645190
17.

Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India.

Nagral A, Mewawalla P, Jagadeesh S, Kabra M, Phadke SR, Verma IC, Puri RD, Gupta N, Kishnani PS, Mistry PK.

Indian Pediatr. 2011 Oct;48(10):779-84.

PMID:
22080680
18.

Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease.

Elstein D, Mehta A, Hughes DA, Giraldo P, Charrow J, Smith L, Shankar SP, Hangartner TN, Kunes Y, Wang N, Crombez E, Zimran A.

Am J Hematol. 2015 Jul;90(7):592-7. doi: 10.1002/ajh.24007.

19.

Evaluation of Spanish Gaucher disease patients after a 6-month imiglucerase shortage.

Giraldo P, Irún P, Alfonso P, Dalmau J, Fernández-Galán MA, Figueredo A, Hernández-Rivas JM, Julia A, Luño E, Marín-Jimenez F, Martín-Nuñez G, Montserrat JL, de la Serna J, Vidaller A, Villalón L, Pocovi M.

Blood Cells Mol Dis. 2011 Jan 15;46(1):115-8. doi: 10.1016/j.bcmd.2010.09.005. Epub 2010 Oct 8.

PMID:
20934891
20.

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. doi: 10.1186/s13023-015-0275-0.

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