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1975 1
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1988 3
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1990 2
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1993 1
1994 1
1995 1
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2001 4
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2004 2
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96 results

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Page 1
Fabry disease revisited: Management and treatment recommendations for adult patients.
Ortiz A, Germain DP, Desnick RJ, Politei J, Mauer M, Burlina A, Eng C, Hopkin RJ, Laney D, Linhart A, Waldek S, Wallace E, Weidemann F, Wilcox WR. Ortiz A, et al. Among authors: waldek s. Mol Genet Metab. 2018 Apr;123(4):416-427. doi: 10.1016/j.ymgme.2018.02.014. Epub 2018 Feb 28. Mol Genet Metab. 2018. PMID: 29530533 Free article. Review.
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: waldek s. 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.
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.
Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tøndel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG. Wallace EL, et al. Among authors: waldek s. J Med Genet. 2024 May 21;61(6):520-530. doi: 10.1136/jmg-2023-109445. J Med Genet. 2024. PMID: 37940383 Free PMC article. Clinical Trial.
Agalsidase-beta therapy for advanced Fabry disease: a randomized trial.
Banikazemi M, Bultas J, Waldek S, Wilcox WR, Whitley CB, McDonald M, Finkel R, Packman S, Bichet DG, Warnock DG, Desnick RJ; Fabry Disease Clinical Trial Study Group. Banikazemi M, et al. Among authors: waldek s. Ann Intern Med. 2007 Jan 16;146(2):77-86. doi: 10.7326/0003-4819-146-2-200701160-00148. Epub 2006 Dec 18. Ann Intern Med. 2007. PMID: 17179052 Free article. Clinical Trial.
It is time to review how unlicensed medicines are used.
Sutherland A, Waldek S. Sutherland A, et al. Among authors: waldek s. Eur J Clin Pharmacol. 2015 Sep;71(9):1029-35. doi: 10.1007/s00228-015-1886-z. Epub 2015 Jul 9. Eur J Clin Pharmacol. 2015. PMID: 26153083 Free PMC article. Review.
A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies.
Holida M, Linhart A, Pisani A, Longo N, Eyskens F, Goker-Alpan O, Wallace E, Deegan P, Tøndel C, Feldt-Rasmussen U, Hughes D, Sakov A, Rocco R, Almon EB, Alon S, Chertkoff R, Warnock DG, Waldek S, Wilcox WR, Bernat JA. Holida M, et al. Among authors: waldek s. J Inherit Metab Dis. 2025 Jan;48(1):e12795. doi: 10.1002/jimd.12795. Epub 2024 Oct 9. J Inherit Metab Dis. 2025. PMID: 39381863 Free PMC article. Clinical Trial.
Enzyme replacement therapy for Fabry's disease.
Waldek S, Germain DP, Wanner C, Warnock DG. Waldek S, et al. Lancet. 2010 May 1;375(9725):1523; author reply 1523-4. doi: 10.1016/S0140-6736(10)60653-8. Lancet. 2010. PMID: 20435225 No abstract available.
Fabry disease: a review of current management strategies.
Mehta A, Beck M, Eyskens F, Feliciani C, Kantola I, Ramaswami U, Rolfs A, Rivera A, Waldek S, Germain DP. Mehta A, et al. Among authors: waldek s. QJM. 2010 Sep;103(9):641-59. doi: 10.1093/qjmed/hcq117. Epub 2010 Jul 21. QJM. 2010. PMID: 20660166 Review.
96 results