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230 results

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Page 1
Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry.
Germain DP, Weidemann F, Abiose A, Patel MR, Cizmarik M, Cole JA, Beitner-Johnson D, Benistan K, Cabrera G, Charrow J, Kantola I, Linhart A, Nicholls K, Niemann M, Scott CR, Sims K, Waldek S, Warnock DG, Strotmann J; Fabry Registry. Germain DP, et al. Among authors: scott cr. Genet Med. 2013 Dec;15(12):958-65. doi: 10.1038/gim.2013.53. Epub 2013 May 23. Genet Med. 2013. PMID: 23703683 Free article.
Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.
Germain DP, Hughes DA, Nicholls K, Bichet DG, Giugliani R, Wilcox WR, Feliciani C, Shankar SP, Ezgu F, Amartino H, Bratkovic D, Feldt-Rasmussen U, Nedd K, Sharaf El Din U, Lourenco CM, Banikazemi M, Charrow J, Dasouki M, Finegold D, Giraldo P, Goker-Alpan O, Longo N, Scott CR, Torra R, Tuffaha A, Jovanovic A, Waldek S, Packman S, Ludington E, Viereck C, Kirk J, Yu J, Benjamin ER, Johnson F, Lockhart DJ, Skuban N, Castelli J, Barth J, Barlow C, Schiffmann R. Germain DP, et al. Among authors: scott cr. N Engl J Med. 2016 Aug 11;375(6):545-55. doi: 10.1056/NEJMoa1510198. N Engl J Med. 2016. PMID: 27509102 Free article. Clinical Trial.
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. Andersson HC, et al. Among authors: scott cr. Genet Med. 2005 Feb;7(2):105-10. doi: 10.1097/01.gim.0000153660.88672.3c. Genet Med. 2005. PMID: 15714077 Free article. Review.
Long-term treatment outcomes in Gaucher disease.
Charrow J, Scott CR. Charrow J, et al. Among authors: scott cr. Am J Hematol. 2015 Jul;90 Suppl 1:S19-24. doi: 10.1002/ajh.24056. Am J Hematol. 2015. PMID: 26096742 Free article. Review.
Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.
Mistry PK, Batista JL, Andersson HC, Balwani M, Burrow TA, Charrow J, Kaplan P, Khan A, Kishnani PS, Kolodny EH, Rosenbloom B, Scott CR, Weinreb N. Mistry PK, et al. Among authors: scott cr. Am J Hematol. 2017 Sep;92(9):929-939. doi: 10.1002/ajh.24801. Epub 2017 Jul 7. Am J Hematol. 2017. PMID: 28569047 Free PMC article. Clinical Trial.
230 results