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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: clarke jt. 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.
Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease.
Ries M, Clarke JT, Whybra C, Timmons M, Robinson C, Schlaggar BL, Pastores G, Lien YH, Kampmann C, Brady RO, Beck M, Schiffmann R. Ries M, et al. Among authors: clarke jt. Pediatrics. 2006 Sep;118(3):924-32. doi: 10.1542/peds.2005-2895. Pediatrics. 2006. PMID: 16950982 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: clarke jt. 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.
A validated disease severity scoring system for Fabry disease.
Giannini EH, Mehta AB, Hilz MJ, Beck M, Bichet DG, Brady RO, West M, Germain DP, Wanner C, Waldek S, Clarke JT, Mengel E, Strotmann JM, Warnock DG, Linhart A. Giannini EH, et al. Among authors: clarke jt. Mol Genet Metab. 2010 Mar;99(3):283-90. doi: 10.1016/j.ymgme.2009.10.178. Epub 2009 Oct 30. Mol Genet Metab. 2010. PMID: 19951842
311 results