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Items: 1 to 20 of 157

1.

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
2.

Impact of imiglucerase supply shortage on clinical and laboratory parameters in Norrbottnian patients with Gaucher disease type 3.

Machaczka M, Kämpe Björkvall C, Wieremiejczyk J, Paucar Arce M, Myhr-Eriksson K, Klimkowska M, Hägglund H, Svenningsson P.

Arch Immunol Ther Exp (Warsz). 2015 Feb;63(1):65-71. doi: 10.1007/s00005-014-0308-8. Epub 2014 Sep 10.

3.

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.

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.

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
6.

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.

7.

Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply.

Zimran A, Altarescu G, Elstein D.

Blood Cells Mol Dis. 2011 Jan 15;46(1):111-4. doi: 10.1016/j.bcmd.2010.05.001. Epub 2010 Jun 9.

PMID:
20542712
8.

Effects of switching from a reduced dose imiglucerase to velaglucerase in type 1 Gaucher disease: clinical and biochemical outcomes.

van Dussen L, Cox TM, Hendriks EJ, Morris E, Akkerman EM, Maas M, Groener JE, Aerts JM, Deegan PB, Hollak CE.

Haematologica. 2012 Dec;97(12):1850-4. doi: 10.3324/haematol.2011.059071. Epub 2012 Jul 6.

9.

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.

10.

Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.

Elstein D, Dweck A, Attias D, Hadas-Halpern I, Zevin S, Altarescu G, Aerts JF, van Weely S, Zimran A.

Blood. 2007 Oct 1;110(7):2296-301. Epub 2007 Jul 3.

11.

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.

12.

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.

13.

[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
14.

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;88(3):172-8. doi: 10.1002/ajh.23383. Epub 2013 Jan 22.

15.

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;53(4):253-60. doi: 10.1016/j.bcmd.2014.05.004. Epub 2014 Jun 18.

PMID:
24950666
16.

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.

17.

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. doi: 10.1016/j.bcmd.2011.09.009. Epub 2011 Nov 1.

PMID:
22047948
18.

Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.

Hollak CE, vom Dahl S, Aerts JM, Belmatoug N, Bembi B, Cohen Y, Collin-Histed T, Deegan P, van Dussen L, Giraldo P, Mengel E, Michelakakis H, Manuel J, Hrebicek M, Parini R, Reinke J, di Rocco M, Pocovi M, Sa Miranda MC, Tylki-Szymanska A, Zimran A, Cox TM.

Blood Cells Mol Dis. 2010 Jan 15;44(1):41-7. doi: 10.1016/j.bcmd.2009.09.006. Epub 2009 Oct 4.

PMID:
19804996
19.

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;94(8):647-51.

PMID:
15352589
20.

Enzyme replacement therapy "drug holiday": results from an unexpected shortage of an orphan drug supply in Australia.

Goldblatt J, Fletcher JM, McGill J, Szer J, Wilson M.

Blood Cells Mol Dis. 2011 Jan 15;46(1):107-10. doi: 10.1016/j.bcmd.2010.05.002. Epub 2010 Jun 1.

PMID:
20684886

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