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22. [Enzyme replacement therapy in type 1 Gaucher's disease]. Aggio MC, Fernández V, Marcilese M. Medicina (B Aires); 1994 May; 54(2):140-4. PubMed ID: 7997130 [Abstract] [Full Text] [Related]
23. Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies. Pastores GM, Elstein D, Hrebícek M, Zimran A. Clin Ther; 2007 Aug; 29(8):1645-54. PubMed ID: 17919546 [Abstract] [Full Text] [Related]
24. Improvement of life quality measured by Lansky Score after enzymatic replacement therapy in children with Gaucher disease type 1. Cerón-Rodríguez M, Barajas-Colón E, Ramírez-Devars L, Gutiérrez-Camacho C, Salgado-Loza JL. Mol Genet Genomic Med; 2018 Jan; 6(1):27-34. PubMed ID: 29471591 [Abstract] [Full Text] [Related]
25. Individualization of long-term enzyme replacement therapy for Gaucher disease. Andersson HC, Charrow J, Kaplan P, Mistry P, Pastores GM, Prakash-Cheng A, Rosenbloom BE, Scott CR, Wappner RS, Weinreb NJ, International Collaborative Gaucher Group U.S. Regional Coordinators. Genet Med; 2005 Feb; 7(2):105-10. PubMed ID: 15714077 [Abstract] [Full Text] [Related]
26. Long term effects of enzyme replacement therapy in an Italian cohort of type 3 Gaucher patients. Sechi A, Deroma L, Dardis A, Ciana G, Bertin N, Concolino D, Linari S, Perria C, Bembi B. Mol Genet Metab; 2014 Nov; 113(3):213-8. PubMed ID: 25127542 [Abstract] [Full Text] [Related]
27. Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships. Hollak CE, de Fost M, van Dussen L, Vom Dahl S, Aerts JM. Expert Opin Pharmacother; 2009 Nov; 10(16):2641-52. PubMed ID: 19743939 [Abstract] [Full Text] [Related]
28. [Gaucher disease type I diagnosed at 63 years old presenting with thrombocytopenia]. Kawahara S, Kato A, Oshimi K, Ida H. Rinsho Ketsueki; 2008 May; 49(5):335-9. PubMed ID: 18572811 [Abstract] [Full Text] [Related]
29. Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme. Hollak CE, Hughes D, van Schaik IN, Schwierin B, Bembi B. Pharmacoepidemiol Drug Saf; 2009 Sep; 18(9):770-7. PubMed ID: 19507165 [Abstract] [Full Text] [Related]
30. 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 Sep; 120(1-2):47-56. PubMed ID: 28040394 [Abstract] [Full Text] [Related]
31. 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; 117(2):164-71. PubMed ID: 26043810 [Abstract] [Full Text] [Related]
32. Coagulation abnormalities in type 1 Gaucher disease in children. Deghady A, Marzouk I, El-Shayeb A, Wali Y. Pediatr Hematol Oncol; 2006 Feb; 23(5):411-7. PubMed ID: 16728361 [Abstract] [Full Text] [Related]
33. Consequences of diagnostic delays in type 1 Gaucher disease: the need for greater awareness among hematologists-oncologists and an opportunity for early diagnosis and intervention. Mistry PK, Sadan S, Yang R, Yee J, Yang M. Am J Hematol; 2007 Aug; 82(8):697-701. PubMed ID: 17492645 [Abstract] [Full Text] [Related]
34. Clinical and enzyme studies in Gaucher disease. Kaur M, Kabra M, Kher A, Naik G, Bharucha BA, Verma IC. Indian Pediatr; 1996 Sep; 33(9):735-8. PubMed ID: 9057400 [Abstract] [Full Text] [Related]
35. Enzyme therapy in Gaucher disease type 1: dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months. Pastores GM, Sibille AR, Grabowski GA. Blood; 1993 Jul 15; 82(2):408-16. PubMed ID: 8392397 [Abstract] [Full Text] [Related]
36. [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. PubMed ID: 9139464 [Abstract] [Full Text] [Related]
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38. Baseline characteristics and outcome in Romanian patients with Gaucher disease type 1. Grigorescu-Sido P, Drugan C, Alkhzouz C, Zimmermann A, Coldea C, Denes C, Grigorescu MD, Cret V, Bucerzan S. Eur J Intern Med; 2010 Apr 14; 21(2):104-13. PubMed ID: 20206881 [Abstract] [Full Text] [Related]
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