629 related articles for article (PubMed ID: 29982630)
21. A synthetic chaperone corrects the trafficking defect and disease phenotype in a protein misfolding disorder.
Yam GH; Zuber C; Roth J
FASEB J; 2005 Jan; 19(1):12-8. PubMed ID: 15629890
[TBL] [Abstract][Full Text] [Related]
22. Precision medicine in Fabry disease.
Lenders M; Brand E
Nephrol Dial Transplant; 2021 Jun; 36(Suppl 2):14-23. PubMed ID: 34153986
[TBL] [Abstract][Full Text] [Related]
23. Increased globotriaosylceramide levels in a transgenic mouse expressing human alpha1,4-galactosyltransferase and a mouse model for treating Fabry disease.
Shiozuka C; Taguchi A; Matsuda J; Noguchi Y; Kunieda T; Uchio-Yamada K; Yoshioka H; Hamanaka R; Yano S; Yokoyama S; Mannen K; Kulkarni AB; Furukawa K; Ishii S
J Biochem; 2011 Feb; 149(2):161-70. PubMed ID: 20961863
[TBL] [Abstract][Full Text] [Related]
24. Generation of Fabry cardiomyopathy model for drug screening using induced pluripotent stem cell-derived cardiomyocytes from a female Fabry patient.
Kuramoto Y; Naito AT; Tojo H; Sakai T; Ito M; Shibamoto M; Nakagawa A; Higo T; Okada K; Yamaguchi T; Lee JK; Miyagawa S; Sawa Y; Sakata Y; Komuro I
J Mol Cell Cardiol; 2018 Aug; 121():256-265. PubMed ID: 30048710
[TBL] [Abstract][Full Text] [Related]
25. Fabry Disease: The Current Treatment Landscape.
Lenders M; Brand E
Drugs; 2021 Apr; 81(6):635-645. PubMed ID: 33721270
[TBL] [Abstract][Full Text] [Related]
26. Mutation-specific Fabry disease patient-derived cell model to evaluate the amenability to chaperone therapy.
Lenders M; Stappers F; Niemietz C; Schmitz B; Boutin M; Ballmaier PJ; Zibert A; Schmidt H; Brand SM; Auray-Blais C; Brand E
J Med Genet; 2019 Aug; 56(8):548-556. PubMed ID: 31010832
[TBL] [Abstract][Full Text] [Related]
27. Synergy between the pharmacological chaperone 1-deoxygalactonojirimycin and the human recombinant alpha-galactosidase A in cultured fibroblasts from patients with Fabry disease.
Porto C; Pisani A; Rosa M; Acampora E; Avolio V; Tuzzi MR; Visciano B; Gagliardo C; Materazzi S; la Marca G; Andria G; Parenti G
J Inherit Metab Dis; 2012 May; 35(3):513-20. PubMed ID: 22187137
[TBL] [Abstract][Full Text] [Related]
28. A Phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects.
Giugliani R; Waldek S; Germain DP; Nicholls K; Bichet DG; Simosky JK; Bragat AC; Castelli JP; Benjamin ER; Boudes PF
Mol Genet Metab; 2013 May; 109(1):86-92. PubMed ID: 23474038
[TBL] [Abstract][Full Text] [Related]
29. Fabry Nephropathy: An Evidence-Based Narrative Review.
Del Pino M; Andrés A; Bernabéu AÁ; de Juan-Rivera J; Fernández E; de Dios García Díaz J; Hernández D; Luño J; Fernández IM; Paniagua J; Posada de la Paz M; Rodríguez-Pérez JC; Santamaría R; Torra R; Ambros JT; Vidau P; Torregrosa JV
Kidney Blood Press Res; 2018; 43(2):406-421. PubMed ID: 29558749
[TBL] [Abstract][Full Text] [Related]
30. Distributions of Globotriaosylceramide Isoforms, and Globotriaosylsphingosine and Its Analogues in an α-Galactosidase A Knockout Mouse, a Model of Fabry Disease.
Sueoka H; Aoki M; Tsukimura T; Togawa T; Sakuraba H
PLoS One; 2015; 10(12):e0144958. PubMed ID: 26661087
[TBL] [Abstract][Full Text] [Related]
31. An in vitro model of Fabry disease.
Shu L; Murphy HS; Cooling L; Shayman JA
J Am Soc Nephrol; 2005 Sep; 16(9):2636-45. PubMed ID: 16033856
[TBL] [Abstract][Full Text] [Related]
32. Impact of the organic cation transporter 2 inhibitor cimetidine on the single-dose pharmacokinetics of the glucosylceramide synthase inhibitor lucerastat in healthy subjects.
Boof ML; Halabi A; Ufer M; Dingemanse J
Eur J Clin Pharmacol; 2020 Mar; 76(3):431-437. PubMed ID: 31836927
[TBL] [Abstract][Full Text] [Related]
33. 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
N Engl J Med; 2016 Aug; 375(6):545-55. PubMed ID: 27509102
[TBL] [Abstract][Full Text] [Related]
34. Differences in cleavage of globotriaosylceramide and its derivatives accumulated in organs of young Fabry mice following enzyme replacement therapy.
Kodama T; Tsukimura T; Kawashima I; Sato A; Sakuraba H; Togawa T
Mol Genet Metab; 2017; 120(1-2):116-120. PubMed ID: 27756537
[TBL] [Abstract][Full Text] [Related]
35. The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in Fabry patient cell lines.
Benjamin ER; Flanagan JJ; Schilling A; Chang HH; Agarwal L; Katz E; Wu X; Pine C; Wustman B; Desnick RJ; Lockhart DJ; Valenzano KJ
J Inherit Metab Dis; 2009 Jun; 32(3):424-40. PubMed ID: 19387866
[TBL] [Abstract][Full Text] [Related]
36. Molecular basis of 1-deoxygalactonojirimycin arylthiourea binding to human α-galactosidase a: pharmacological chaperoning efficacy on Fabry disease mutants.
Yu Y; Mena-Barragán T; Higaki K; Johnson JL; Drury JE; Lieberman RL; Nakasone N; Ninomiya H; Tsukimura T; Sakuraba H; Suzuki Y; Nanba E; Mellet CO; García Fernández JM; Ohno K
ACS Chem Biol; 2014 Jul; 9(7):1460-9. PubMed ID: 24783948
[TBL] [Abstract][Full Text] [Related]
37. Pharmacological chaperone corrects lysosomal storage in Fabry disease caused by trafficking-incompetent variants.
Yam GH; Bosshard N; Zuber C; Steinmann B; Roth J
Am J Physiol Cell Physiol; 2006 Apr; 290(4):C1076-82. PubMed ID: 16531566
[TBL] [Abstract][Full Text] [Related]
38. The New Pharmacological Chaperones PBXs Increase α-Galactosidase A Activity in Fabry Disease Cellular Models.
Besada P; Gallardo-Gómez M; Pérez-Márquez T; Patiño-Álvarez L; Pantano S; Silva-López C; Terán C; Arévalo-Gómez A; Ruz-Zafra A; Fernández-Martín J; Ortolano S
Biomolecules; 2021 Dec; 11(12):. PubMed ID: 34944500
[TBL] [Abstract][Full Text] [Related]
39. Treatment of fabry disease: current and emerging strategies.
Rozenfeld P; Neumann PM
Curr Pharm Biotechnol; 2011 Jun; 12(6):916-22. PubMed ID: 21235448
[TBL] [Abstract][Full Text] [Related]
40. Urinary globotriaosylceramide excretion correlates with the genotype in children and adults with Fabry disease.
Auray-Blais C; Cyr D; Ntwari A; West ML; Cox-Brinkman J; Bichet DG; Germain DP; Laframboise R; Melançon SB; Stockley T; Clarke JT; Drouin R
Mol Genet Metab; 2008 Mar; 93(3):331-40. PubMed ID: 18023222
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
