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6. Novel heterozygous c.798C>G and c.1040T>G mutations in the GBA1 gene are associated with a severe phenotype of Gaucher disease type 1. Machaczka M; Klimkowska M Ann Hematol; 2014 Oct; 93(10):1787-9. PubMed ID: 24577513 [No Abstract] [Full Text] [Related]
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; 120(1-2):47-56. PubMed ID: 28040394 [TBL] [Abstract][Full Text] [Related]
9. IgM monoclonal component associated with type I Gaucher disease resolved after enzyme replacement therapy: a case report. Martinez-Redondo C; Ortuño FJ; Lozano ML; Jerez A; del Mar Osma M; Giraldo P; Vicente V J Inherit Metab Dis; 2009 Dec; 32 Suppl 1():S265-7. PubMed ID: 19562501 [TBL] [Abstract][Full Text] [Related]
10. Evolving features in type 3 Gaucher disease on long-term enzyme replacement therapy. Elstein D; Abrahamov A; Altarescu G; Zimran A Blood Cells Mol Dis; 2013 Feb; 50(2):140. PubMed ID: 23085428 [No Abstract] [Full Text] [Related]
11. Therapy of adult Gaucher disease. Schmitz J; Poll LW; vom Dahl S Haematologica; 2007 Feb; 92(2):148-52. PubMed ID: 17296562 [No Abstract] [Full Text] [Related]
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13. Velaglucerase alfa, a human recombinant glucocerebrosidase enzyme replacement therapy for type 1 Gaucher disease. Pastores GM Curr Opin Investig Drugs; 2010 Apr; 11(4):472-8. PubMed ID: 20336596 [TBL] [Abstract][Full Text] [Related]
15. Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations. Charrow J; Andersson HC; Kaplan P; Kolodny EH; Mistry P; Pastores G; Prakash-Cheng A; Rosenbloom BE; Scott CR; Wappner RS; Weinreb NJ J Pediatr; 2004 Jan; 144(1):112-20. PubMed ID: 14722528 [No Abstract] [Full Text] [Related]
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18. Effects of switching from imiglucerase to velaglucerase alfa without dose reduction nor wash out in type 1 Gaucher disease. Serratrice C; Bengherbia M; Alessandrini M; Grosbois B; Camou F; Pers YM; Bismuth M; Marie I; Belmatoug N; Berger M; Blood Cells Mol Dis; 2014; 53(1-2):94-6. PubMed ID: 24411065 [No Abstract] [Full Text] [Related]
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