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Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment.
Weinreb NJ, Camelo JS Jr, Charrow J, McClain MR, Mistry P, Belmatoug N; International Collaborative Gaucher Group (ICGG) Gaucher Registry (NCT00358943) investigators. Weinreb NJ, et al. Mol Genet Metab. 2021 Feb;132(2):100-111. doi: 10.1016/j.ymgme.2020.12.295. Epub 2021 Jan 8. Mol Genet Metab. 2021. PMID: 33485799 Free article.
Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients.
Weinreb NJ, Aggio MC, Andersson HC, Andria G, Charrow J, Clarke JT, Erikson A, Giraldo P, Goldblatt J, Hollak C, Ida H, Kaplan P, Kolodny EH, Mistry P, Pastores GM, Pires R, Prakash-Cheng A, Rosenbloom BE, Scott CR, Sobreira E, Tylki-Szymańska A, Vellodi A, vom Dahl S, Wappner RS, Zimran A; International Collaborative Gaucher Group (ICGG). Weinreb NJ, et al. Semin Hematol. 2004 Oct;41(4 Suppl 5):15-22. doi: 10.1053/j.seminhematol.2004.07.010. Semin Hematol. 2004. PMID: 15468046