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8. Fabry disease genotype, phenotype, and migalastat amenability: Insights from a national cohort. Nowak A; Huynh-Do U; Krayenbuehl PA; Beuschlein F; Schiffmann R; Barbey F J Inherit Metab Dis; 2020 Mar; 43(2):326-333. PubMed ID: 31449323 [TBL] [Abstract][Full Text] [Related]
9. 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 Mol Genet Metab; 2020; 131(1-2):219-228. PubMed ID: 33012654 [TBL] [Abstract][Full Text] [Related]
10. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Benjamin ER; Della Valle MC; Wu X; Katz E; Pruthi F; Bond S; Bronfin B; Williams H; Yu J; Bichet DG; Germain DP; Giugliani R; Hughes D; Schiffmann R; Wilcox WR; Desnick RJ; Kirk J; Barth J; Barlow C; Valenzano KJ; Castelli J; Lockhart DJ Genet Med; 2017 Apr; 19(4):430-438. PubMed ID: 27657681 [TBL] [Abstract][Full Text] [Related]
11. Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study. Germain DP; Nicholls K; Giugliani R; Bichet DG; Hughes DA; Barisoni LM; Colvin RB; Jennette JC; Skuban N; Castelli JP; Benjamin E; Barth JA; Viereck C Genet Med; 2019 Sep; 21(9):1987-1997. PubMed ID: 30723321 [TBL] [Abstract][Full Text] [Related]
12. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Narita I; Ohashi T; Sakai N; Hamazaki T; Skuban N; Castelli JP; Lagast H; Barth JA Clin Exp Nephrol; 2020 Feb; 24(2):157-166. PubMed ID: 31889231 [TBL] [Abstract][Full Text] [Related]
13. Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable Mauer M; Sokolovskiy A; Barth JA; Castelli JP; Williams HN; Benjamin ER; Najafian B J Med Genet; 2017 Nov; 54(11):781-786. PubMed ID: 28756410 [TBL] [Abstract][Full Text] [Related]
14. 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]
15. Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS). Lenders M; Nordbeck P; Kurschat C; Karabul N; Kaufeld J; Hennermann JB; Patten M; Cybulla M; Müntze J; Üçeyler N; Liu D; Das AM; Sommer C; Pogoda C; Reiermann S; Duning T; Gaedeke J; Stumpfe K; Blaschke D; Brand SM; Mann WA; Kampmann C; Muschol N; Canaan-Kühl S; Brand E Clin Pharmacol Ther; 2020 Aug; 108(2):326-337. PubMed ID: 32198894 [TBL] [Abstract][Full Text] [Related]
16. Challenges in Fabry disease: the combination of two individually amenable Fernandes RM; Bento D; Marques N; Azevedo O; Mota T; Costa H; Santo ME; Silva DC; Jesus I Future Cardiol; 2023 Jan; 19(1):39-43. PubMed ID: 36695159 [TBL] [Abstract][Full Text] [Related]
17. A review and recommendations for oral chaperone therapy in adult patients with Fabry disease. Nowicki M; Bazan-Socha S; Błażejewska-Hyżorek B; Kłopotowski MM; Komar M; Kusztal MA; Liberek T; Małyszko J; Mizia-Stec K; Oko-Sarnowska Z; Pawlaczyk K; Podolec P; Sławek J; Orphanet J Rare Dis; 2024 Jan; 19(1):16. PubMed ID: 38238782 [TBL] [Abstract][Full Text] [Related]
18. Strong increase of leukocyte apha-galactosidase A activity in two male patients with Fabry disease following oral chaperone therapy. Lamari F; Mauhin W; Koraichi F; Khrouf W; Bordet C; London J; Lidove O; Charron P Mol Genet Genomic Med; 2019 Sep; 7(9):e894. PubMed ID: 31393666 [TBL] [Abstract][Full Text] [Related]
19. A Systematic Review on Safety and Efficacy of Migalastat for the treatment of Fabry's Disease. Majid H; Verma N; Bhandari S; Gupta S; Nidhi Expert Opin Pharmacother; 2024 Apr; 25(6):769-782. PubMed ID: 38753367 [TBL] [Abstract][Full Text] [Related]
20. Treatment of Fabry Disease management with migalastat-outcome from a prospective 24 months observational multicenter study (FAMOUS). Lenders M; Nordbeck P; Kurschat C; Eveslage M; Karabul N; Kaufeld J; Hennermann JB; Patten M; Cybulla M; Müntze J; Üçeyler N; Liu D; Das AM; Sommer C; Pogoda C; Reiermann S; Duning T; Gaedeke J; von Cossel K; Blaschke D; Brand SM; Mann WA; Kampmann C; Muschol N; Canaan-Kühl S; Brand E Eur Heart J Cardiovasc Pharmacother; 2022 May; 8(3):272-281. PubMed ID: 35512362 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]