Long-Term Efficacy of Migalastat on Renal Function and Outcomes in Patients with Fabry Disease (FD)

被引：0
|
作者：
Bichet, Daniel G. [1 ]
Feldt-Rasmussen, Ulla [2 ]
Giugliani, Roberto [3 ,4 ]
Hughes, Derralynn [5 ,6 ]
Krusinska, Eva [7 ]
Veleva-Rotse, Biliana O. [7 ]
机构：
[1] Univ Montreal, Hop Sacre Coeur, Dept Med, Montreal, PQ, Canada
[2] Natl Univ Hosp, Rigshosp, Dept Med Endocrinol & Metab, Copenhagen, Denmark
[3] Univ Fed Rio Grande do Sul, Dept Genet, HCPA, Med Genet Serv, Porto Alegre, RS, Brazil
[4] INAGEMP, Porto Alegre, RS, Brazil
[5] Royal Free London NHS Fdn Trust, Lysosomal Storage Disorders Unit, London, England
[6] UCL, London, England
[7] Amicus Therapeut Inc, Philadelphia, PA USA
来源：
JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY | 2021年 / 32卷 / 10期
关键词：
D O I：
暂无
中图分类号：
R5 [内科学]; R69 [泌尿科学（泌尿生殖系疾病）];
学科分类号：
1002 ; 100201 ;
摘要：
PO1312
引用
收藏
页码：420 / 421
页数：2
相关论文
共 50 条
  • [41] Sustained, long-term renal stabilization after 54 months of agalsidase β therapy in patients with Fabry disease
    Germain, Dominique P.
    Waldek, Stephen
    Banikazemi, Maryam
    Bushinsky, David A.
    Charrow, Joel
    Desnick, Robert J.
    Lee, Philip
    Loew, Thomas
    Vedder, Anouk C.
    Abichandani, Rekha
    Wilcox, William R.
    Guffon, Nathalie
    JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2007, 18 (05): : 1547 - 1557
  • [42] Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting
    Schiffmann, R
    Ries, M
    Timmons, M
    Flaherty, JT
    Brady, RO
    NEPHROLOGY DIALYSIS TRANSPLANTATION, 2006, 21 (02) : 345 - 354
  • [43] Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing
    Schiffmann, Raphael
    Askari, Hasan
    Timmons, Margaret
    Robinson, Chevalia
    Benko, William
    Brady, Roscoe O.
    Ries, Markus
    JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2007, 18 (05): : 1576 - 1583
  • [44] A Systematic Review on Safety and Efficacy of Migalastat for the treatment of Fabry's Disease
    Majid, Haya
    Verma, Neharika
    Bhandari, Shivani
    Gupta, Sparsh
    Nidhi
    EXPERT OPINION ON PHARMACOTHERAPY, 2024, 25 (06) : 769 - 782
  • [45] Long-term renal function in adult patients with phenylketonuria
    Prepok, Flora Franciska
    Schnabel, Karolina Kornelia
    Sumanszki, Csaba
    Barta, Andras Gellert
    Tisler, Andras
    Reismann, Peter
    NEPHRON, 2024, 148 (04) : 195 - 203
  • [46] Biomarkers for Monitoring Renal Damage Due to Fabry Disease in Patients Treated with Migalastat: A Review for Nephrologists
    Jaurretche, Sebastian
    Conde, Hernan
    Schain, Ana Gonzalez
    Ruiz, Franco
    Sgro, Maria Victoria
    Venera, Graciela
    GENES, 2022, 13 (10)
  • [47] Renal outcomes with up to 9 years of migalastat in patients with Fabry disease: Results from an open-label extension study
    Nicholls, Kathleen
    Giugliani, Roberto
    Schiffmann, Raphael
    Hughes, Derralynn A.
    Jain, Vipul
    Holdbrook, Fred
    Skuban, Nina
    Castelli, Jeffery P.
    Barth, Jay A.
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S105 - S106
  • [48] A long-term neuropsychological evaluation in Fabry disease
    Loret, G.
    Miatton, M.
    Vingerhoets, G.
    Poppe, B.
    Hemelsoet, D.
    ACTA NEUROLOGICA BELGICA, 2021, 121 (01) : 191 - 197
  • [49] α-galactosidase activity increases after short-term treatment with migalastat in patients with Fabry disease
    Nowak, Albina
    Desnick, Robert J.
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S107 - S108
  • [50] A long-term neuropsychological evaluation in Fabry disease
    G. Loret
    M. Miatton
    G. Vingerhoets
    B. Poppe
    D. Hemelsoet
    Acta Neurologica Belgica, 2021, 121 : 191 - 197
12345