Long-term follow-up of renal function in patients treated with migalastat for Fabry disease.
Chaperone
Classic phenotype
Efficacy
Fabry disease
GLP-HEK, Good Laboratory Practice-validated human embryonic kidney
Gb3, globotriaosylceramide
LVMi, left ventricular mass index
Migalastat
Q1, quartile 1
Q3, quartile 3
RI, renin inhibitor
Renal function
eGFRCKD-EPI, estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation
α-Gal A, α-galactosidase A
Journal
Molecular genetics and metabolism reports
ISSN: 2214-4269
Titre abrégé: Mol Genet Metab Rep
Pays: United States
ID NLM: 101624422
Informations de publication
Date de publication:
Sep 2021
Sep 2021
Historique:
received:
30
07
2021
accepted:
31
07
2021
entrez:
17
8
2021
pubmed:
18
8
2021
medline:
18
8
2021
Statut:
epublish
Résumé
The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)-naive and ERT-experienced patients with Fabry disease is not well defined. An integrated posthoc analysis of the phase 3 clinical trials and open-label extension studies was conducted to evaluate long-term changes in renal function in patients with Fabry disease and
Identifiants
pubmed: 34401344
doi: 10.1016/j.ymgmr.2021.100786
pii: S2214-4269(21)00080-X
pmc: PMC8353473
doi:
Types de publication
Journal Article
Langues
eng
Pagination
100786Commentaires et corrections
Type : ErratumIn
Informations de copyright
© 2021 The Authors. Published by Elsevier Inc.
Déclaration de conflit d'intérêts
D.G.B. served as a consultant and speaker for and received grants from Amicus, Sanofi Genzyme and Otsuka; and served as a board member for Amicus. E.W. received grants and consulting fees from Sanofi Genzyme and received research and travel support from Protalix and Idorsia. D.H. received consultant fees from Amicus, Sanofi Genzyme, Shire-Takeda, Protalix, Idorisia, and Freeline Therapeutics; and served as a speaker for Amicus, Shire-Takeda, and Sanofi Genzyme. R.G. was a paid consultant for JCR Pharmaceuticals, REGENXBIO, and Sigilon; received grants from BioMarin, Novartis, Shire-Takeda, and Ultragenyx; was a paid speaker for Amicus, BioMarin, Chiesi, Janssen, PTC Therapeutics, Sanofi Genzyme, Shire-Takeda, and Ultragenyx; and served as a board member for Amicus, Abeona, Sobi, Sanofi Genzyme, and Shire-Takeda. N.S. was an employee of Amicus at the time of this study. E.K. served as a paid consultant for Amicus. U.F.R. served as a board member and paid consultant for Amicus, Sanofi Genzyme, and Shire-Takeda, and received an unrestricted research grant from Sanofi Genzyme. K.N. has served on advisory boards for Amicus Therapeutics, Sanofi Genzyme, and Shire; has served as a speaker for Amicus Therapeutics; and has received research funding from Sanofi Genzyme and Shire. R.T. and R.S have no conflicts to declare.
Références
Nephrol Dial Transplant. 2009 Jul;24(7):2102-11
pubmed: 19218538
J Med Genet. 2017 Apr;54(4):288-296
pubmed: 27834756
J Am Soc Nephrol. 2002 Jun;13 Suppl 2:S139-43
pubmed: 12068026
Mol Genet Metab. 2021 Apr;132(4):234-243
pubmed: 33642210
Nefrologia (Engl Ed). 2019 Mar - Apr;39(2):177-183
pubmed: 30139698
Ann Intern Med. 2009 May 5;150(9):604-12
pubmed: 19414839
Nephrol Dial Transplant. 2010 Mar;25(3):769-75
pubmed: 19846394
Lancet. 2009 Dec 12;374(9706):1986-96
pubmed: 19959221
Clin Genet. 2014 Sep;86(3):258-63
pubmed: 23980562
Nephron. 2015;129(1):16-21
pubmed: 25531941
Mol Genet Metab. 2020 Feb;129(2):132-141
pubmed: 31718986
Nephrol Dial Transplant. 2012 Mar;27(3):1042-9
pubmed: 21804088
Nat Clin Pract Nephrol. 2008 Jun;4(6):327-36
pubmed: 18431378
Genet Med. 2017 Apr;19(4):430-438
pubmed: 27657681
Mol Genet Metab. 2016 Feb;117(2):104-13
pubmed: 26546059
J Am Soc Nephrol. 2002 Jun;13 Suppl 2:S134-8
pubmed: 12068025
Ann Intern Med. 2007 Jan 16;146(2):77-86
pubmed: 17179052
N Engl J Med. 2004 Sep 23;351(13):1296-305
pubmed: 15385656
Orphanet J Rare Dis. 2010 Nov 22;5:30
pubmed: 21092187
J Med Genet. 2017 Nov;54(11):781-786
pubmed: 28756410
J Med Genet. 2009 Aug;46(8):548-52
pubmed: 19473999
Eur Heart J Cardiovasc Pharmacother. 2022 May 5;8(3):272-281
pubmed: 35512362
N Engl J Med. 2016 Aug 11;375(6):545-55
pubmed: 27509102
Genet Med. 2019 Sep;21(9):1987-1997
pubmed: 30723321
Stat Med. 1999 Apr 15;18(7):855-88
pubmed: 10327531
Kidney Int. 2011 Mar;79(6):663-670
pubmed: 21160462
Nephrol Dial Transplant. 2019 Sep 1;34(9):1525-1533
pubmed: 30535327
PLoS One. 2015 Jun 10;10(6):e0129036
pubmed: 26061083
Orphanet J Rare Dis. 2015 Mar 27;10:36
pubmed: 25885911
Genet Med. 2009 Nov;11(11):790-6
pubmed: 19745746
Am J Hum Genet. 2004 Jul;75(1):65-74
pubmed: 15154115
Mol Genet Metab Rep. 2015 Mar 05;3:21-7
pubmed: 26937390
J Am Soc Nephrol. 2007 May;18(5):1547-57
pubmed: 17409312
Nephron. 2019;141(1):10-17
pubmed: 30466100
Nephrol Dial Transplant. 2011 Jun;26(6):1797-802
pubmed: 20504837
Biochem J. 2007 Sep 1;406(2):285-95
pubmed: 17555407
J Med Genet. 2015 May;52(5):353-8
pubmed: 25795794
FASEB J. 2005 Jan;19(1):12-8
pubmed: 15629890
J Am Soc Nephrol. 2020 Apr;31(4):865-875
pubmed: 32127409
Clin J Am Soc Nephrol. 2010 Dec;5(12):2220-8
pubmed: 20813854
Orphanet J Rare Dis. 2013 Mar 25;8:47
pubmed: 23531228