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Changes in plasma and urine globotriaosylceramide levels do not predict Fabry disease progression over 1 year of agalsidase alfa.
Schiffmann R, Ries M, Blankenship D, Nicholls K, Mehta A, Clarke JT, Steiner RD, Beck M, Barshop BA, Rhead W, West M, Martin R, Amato D, Nair N, Huertas P. Schiffmann R, et al. Among authors: nicholls k. Genet Med. 2013 Dec;15(12):983-9. doi: 10.1038/gim.2013.56. Epub 2013 May 16. Genet Med. 2013. PMID: 23680766 Free article. Clinical Trial.
Agalsidase alfa and kidney dysfunction in Fabry disease.
West M, Nicholls K, Mehta A, Clarke JT, Steiner R, Beck M, Barshop BA, Rhead W, Mensah R, Ries M, Schiffmann R. West M, et al. Among authors: nicholls k. J Am Soc Nephrol. 2009 May;20(5):1132-9. doi: 10.1681/ASN.2008080870. Epub 2009 Apr 8. J Am Soc Nephrol. 2009. PMID: 19357250 Free PMC article.
Therapeutic goals in the treatment of Fabry disease.
Mehta A, West ML, Pintos-Morell G, Reisin R, Nicholls K, Figuera LE, Parini R, Carvalho LR, Kampmann C, Pastores GM, Lidove O. Mehta A, et al. Among authors: nicholls k. Genet Med. 2010 Nov;12(11):713-20. doi: 10.1097/GIM.0b013e3181f6e676. Genet Med. 2010. PMID: 20975569 Free article.
Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial.
Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, Barisoni L, Colvin RB, Jennette CJ, Maegawa G, Boyadjiev SA, Gonzalez D, Nicholls K, Tuffaha A, Atta MG, Rup B, Charney MR, Paz A, Szlaifer M, Alon S, Brill-Almon E, Chertkoff R, Hughes D. Schiffmann R, et al. Among authors: nicholls k. J Inherit Metab Dis. 2019 May;42(3):534-544. doi: 10.1002/jimd.12080. Epub 2019 Apr 8. J Inherit Metab Dis. 2019. PMID: 30834538 Clinical Trial.
Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis.
Ramaswami U, Beck M, Hughes D, Kampmann C, Botha J, Pintos-Morell G, West ML, Niu DM, Nicholls K, Giugliani R; FOS Study Group. Ramaswami U, et al. Among authors: nicholls k. Drug Des Devel Ther. 2019 Oct 25;13:3705-3715. doi: 10.2147/DDDT.S207856. eCollection 2019. Drug Des Devel Ther. 2019. PMID: 31749608 Free PMC article.
234 results