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Items: 1 to 20 of 1003

1.

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;122(6):1182-90. doi: 10.1542/peds.2007-2144.

PMID:
19047232
2.

Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry.

Weinreb NJ, Charrow J, Andersson HC, Kaplan P, Kolodny EH, Mistry P, Pastores G, Rosenbloom BE, Scott CR, Wappner RS, Zimran A.

Am J Med. 2002 Aug 1;113(2):112-9.

PMID:
12133749
3.

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.

4.

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;151(2):197-201. Epub 2007 Jun 22.

PMID:
17643778
5.

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
6.

Report of the Spanish Gaucher's disease registry: clinical and genetic characteristics.

Giraldo P, Pocoví M, Pérez-Calvo J, Rubio-Félix D, Giralt M.

Haematologica. 2000 Aug;85(8):792-9.

7.

The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease.

Charrow J, Dulisse B, Grabowski GA, Weinreb NJ.

Clin Genet. 2007 Mar;71(3):205-11.

PMID:
17309642
8.

Enzyme replacement therapy for Gaucher disease in Australia.

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

Intern Med J. 2005 Mar;35(3):156-61.

PMID:
15737135
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.

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.

11.

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.

12.

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
13.

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;69(5):228-32.

14.

Acceleration of retarded growth in children with Gaucher disease after treatment with alglucerase.

Kaplan P, Mazur A, Manor O, Charrow J, Esplin J, Gribble TJ, Wappner RS, Wisch JS, Weinreb NJ.

J Pediatr. 1996 Jul;129(1):149-53.

PMID:
8757576
15.

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
16.

[Enzyme replacement therapy: a new treatment concept in Gaucher disease].

Beck M, Mengel E, Barone R.

Wien Klin Wochenschr. 1997 Feb 14;109(3):81-5. German.

PMID:
9139464
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.

[Enzyme replacement therapy in Gaucher disease: monitoring visceral and bone changes with MRI].

Tóth J, Szücs FZ, Benkö K, Maródi L.

Orv Hetil. 2003 Apr 20;144(16):749-55. Hungarian.

PMID:
12778625
19.

Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1.

Grabowski GA, Kacena K, Cole JA, Hollak CE, Zhang L, Yee J, Mistry PK, Zimran A, Charrow J, vom Dahl S.

Genet Med. 2009 Feb;11(2):92-100. doi: 10.1097/GIM.0b013e31818e2c19.

20.

Long-term bone mineral density response to enzyme replacement therapy in a retrospective pediatric cohort of Gaucher patients.

Ciana G, Deroma L, Franzil AM, Dardis A, Bembi B.

J Inherit Metab Dis. 2012 Nov;35(6):1101-6. doi: 10.1007/s10545-012-9476-z. Epub 2012 Mar 23.

PMID:
22441841
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