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Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.
Hughes DA, Nicholls K, Shankar SP, Sunder-Plassmann G, Koeller D, Nedd K, Vockley G, Hamazaki T, Lachmann R, Ohashi T, Olivotto I, Sakai N, Deegan P, Dimmock D, Eyskens F, Germain DP, Goker-Alpan O, Hachulla E, Jovanovic A, Lourenco CM, Narita I, Thomas M, Wilcox WR, Bichet DG, Schiffmann R, Ludington E, Viereck C, Kirk J, Yu J, Johnson F, Boudes P, Benjamin ER, Lockhart DJ, Barlow C, Skuban N, Castelli JP, Barth J, Feldt-Rasmussen U. Hughes DA, et al. Among authors: hamazaki t. J Med Genet. 2017 Apr;54(4):288-296. doi: 10.1136/jmedgenet-2016-104178. Epub 2016 Nov 10. J Med Genet. 2017. PMID: 27834756 Free PMC article. Clinical Trial.
Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study.
Feldt-Rasmussen U, Hughes D, Sunder-Plassmann G, Shankar S, Nedd K, Olivotto I, Ortiz D, Ohashi T, Hamazaki T, Skuban N, Yu J, Barth JA, Nicholls K. Feldt-Rasmussen U, et al. Among authors: hamazaki t. Mol Genet Metab. 2020 Sep-Oct;131(1-2):219-228. doi: 10.1016/j.ymgme.2020.07.007. Epub 2020 Aug 15. Mol Genet Metab. 2020. PMID: 33012654 Free article. Clinical Trial.
Hematopoietic Stem Cell Transplantation for Patients with Mucopolysaccharidosis II.
Kubaski F, Yabe H, Suzuki Y, Seto T, Hamazaki T, Mason RW, Xie L, Onsten TGH, Leistner-Segal S, Giugliani R, Dũng VC, Ngoc CTB, Yamaguchi S, Montaño AM, Orii KE, Fukao T, Shintaku H, Orii T, Tomatsu S. Kubaski F, et al. Among authors: hamazaki t. Biol Blood Marrow Transplant. 2017 Oct;23(10):1795-1803. doi: 10.1016/j.bbmt.2017.06.020. Epub 2017 Jul 1. Biol Blood Marrow Transplant. 2017. PMID: 28673849 Free PMC article.
Divergent developmental trajectories in two siblings with neuropathic mucopolysaccharidosis type II (Hunter syndrome) receiving conventional and novel enzyme replacement therapies: A case report.
Tomita K, Okamoto S, Seto T, Hamazaki T, So S, Yamamoto T, Tanizawa K, Sonoda H, Sato Y. Tomita K, et al. Among authors: hamazaki t. JIMD Rep. 2021 Jul 27;62(1):9-14. doi: 10.1002/jmd2.12239. eCollection 2021 Nov. JIMD Rep. 2021. PMID: 34765392 Free PMC article.
314 results