Fabry disease: A pharmacological chaperone on the horizon.
Division of Nephrology and Dialysis, Department of Medicine III, Medical University of Vienna, Währinger Gürtel 18-20, Vienna 1090, Austria.
Martina
M
Gaggl
Division of Nephrology and Dialysis, Department of Medicine III, Medical University of Vienna, Währinger Gürtel 18-20, Vienna 1090, Austria.
Gere
G
Sunder-Plassmann
D017485:1-Deoxynojirimycin / Q000031:analogs & derivatives; D000795:Fabry Disease; D006801:Humans; D018832:Molecular Chaperones
D018832:Molecular Chaperones; D017485:1-Deoxynojirimycin; C090092:migalastat
N Engl J Med. 2016 Aug 11;375(6):545-55
27509102
Chem Commun (Camb). 2016 Apr 25;52(32):5497-515
27043200
PLoS One. 2015 Aug 07;10 (8):e0134341
26252393
J Am Soc Nephrol. 2016 Jan;27(1):256-64
25933799
J Intern Med. 2013 Oct;274(4):329-30
23662843
Mol Genet Metab. 2008 Sep-Oct;95(1-2):114-5
18701330
Drugs. 2016 Jul;76(11):1147-52
27351440
Orphanet J Rare Dis. 2012 Nov 24;7:91
23176611
Ann Intern Med. 2007 Mar 20;146(6):425-33
17371887