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Diagnosis features of pediatric Gaucher disease patients in the era of enzymatic therapy, a national-base study from the Spanish Registry of Gaucher Disease.

Andrade-Campos M, Alfonso P, Irun P, Armstrong J, Calvo C, Dalmau J, Domingo MR, Barbera JL, Cano H, Fernandez-Galán MA, Franco R, Gracia I, Gracia-Antequera M, Ibañez A, Lendinez F, Madruga M, Martin-Hernández E, O'Callaghan MDM, Del Soto AP, Del Prado YR, Sancho-Val I, Sanjurjo P, Pocovi M, Giraldo P.

Orphanet J Rare Dis. 2017 May 3;12(1):84. doi: 10.1186/s13023-017-0627-z.


Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy.

Serratrice C, Carballo S, Serratrice J, Stirnemann J.

Core Evid. 2016 Oct 14;11:37-47. eCollection 2016. Review.


Gaucher disease in Iraqi children (Clinical, diagnostic & therapeutic aspects).

Thejeal RF, Kadhum AJ.

Pak J Med Sci. 2016 Mar-Apr;32(2):319-23. doi: 10.12669/pjms.322.9316.


Orphan drugs: expensive yet necessary.

Hyry HI, Roos JC, Cox TM.

QJM. 2016 May;109(5):361. doi: 10.1093/qjmed/hcv150. Epub 2015 Sep 3. No abstract available.


Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series.

Serratrice C, Swiader L, Serratrice J.

J Med Case Rep. 2015 Jun 23;9:146. doi: 10.1186/s13256-015-0617-5.


Pathogenesis of Bone Alterations in Gaucher Disease: The Role of Immune System.

Mucci JM, Rozenfeld P.

J Immunol Res. 2015;2015:192761. doi: 10.1155/2015/192761. Epub 2015 May 3. Review.


Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials.

Hughes DA, Gonzalez DE, Lukina EA, Mehta A, Kabra M, Elstein D, Kisinovsky I, Giraldo P, Bavdekar A, Hangartner TN, Wang N, Crombez E, Zimran A.

Am J Hematol. 2015 Jul;90(7):584-91. doi: 10.1002/ajh.24012.


Study of enzyme replacement therapy for Gaucher Disease: comparative analysis of clinical and laboratory parameters at diagnosis and after two, five and ten years of treatment.

Souza AM, Muniz TP, Brito RM.

Rev Bras Hematol Hemoter. 2014 Sep-Oct;36(5):345-50. doi: 10.1016/j.bjhh.2014.05.005. Epub 2014 May 28.


Imaging of Gaucher disease.

Simpson WL, Hermann G, Balwani M.

World J Radiol. 2014 Sep 28;6(9):657-68. doi: 10.4329/wjr.v6.i9.657. Review.


Achievement of therapeutic goals with low-dose imiglucerase in Gaucher disease: a single-center experience.

Tukan I, Hadas-Halpern I, Altarescu G, Abrahamov A, Elstein D, Zimran A.

Adv Hematol. 2013;2013:151506. doi: 10.1155/2013/151506. Epub 2013 Oct 28.


Effects of imiglucerase on the growth and metabolism of Gaucher disease type I patients: a systematic review.

Doneda D, Netto CB, Moulin CC, Schwartz IV.

Nutr Metab (Lond). 2013 Apr 9;10(1):34. doi: 10.1186/1743-7075-10-34.


Bone turnover markers in patients with type 1 Gaucher disease.

Giuffrida G, Cingari MR, Parrinello N, Romano A, Triolo A, Franceschino M, Di Raimondo F.

Hematol Rep. 2012 Nov 19;4(4):e21. doi: 10.4081/hr.2012.e21. Epub 2012 Nov 29.


Imiglucerase in the treatment of Gaucher disease: a history and perspective.

Deegan PB, Cox TM.

Drug Des Devel Ther. 2012;6:81-106. doi: 10.2147/DDDT.S14395. Epub 2012 Apr 18. Review.


Coxarthritis as the presenting symptom of Gaucher disease type 1.

Brisca G, Di Rocco M, Picco P, Damasio MB, Martini A.

Arthritis. 2011;2011:361279. doi: 10.1155/2011/361279. Epub 2011 Mar 30.


Velaglucerase alfa in the treatment of Gaucher disease type 1.

Burrow TA, Grabowski GA.

Clin Investig (Lond). 2011 Feb;1(2):285-293.


Reducing selection bias in case-control studies from rare disease registries.

Cole JA, Taylor JS, Hangartner TN, Weinreb NJ, Mistry PK, Khan A.

Orphanet J Rare Dis. 2011 Sep 12;6:61. doi: 10.1186/1750-1172-6-61.


Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.

Hollak CE, Aerts JM, Aymé S, Manuel J.

Orphanet J Rare Dis. 2011 Apr 16;6:16. doi: 10.1186/1750-1172-6-16. Review.


Gaucher disease: clinical profile and therapeutic developments.

Cox TM.

Biologics. 2010 Dec 6;4:299-313. doi: 10.2147/BTT.S7582.


Optimal therapy in Gaucher disease.

Goker-Alpan O.

Ther Clin Risk Manag. 2010 Jul 21;6:315-23.


Review of the safety and efficacy of imiglucerase treatment of Gaucher disease.

Elstein D, Zimran A.

Biologics. 2009;3:407-17. Epub 2009 Sep 15.

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