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

页码：420 / 421

页数：2

共 50 条

[1] Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
Bichet, Daniel G.
Torra, Roser
Wallace, Eric
Hughes, Derralynn
Giugliani, Roberto
Skuban, Nina
Krusinska, Eva
Feldt-Rasmussen, Ulla
Schiffmann, Raphael
Nicholls, Kathy
MOLECULAR GENETICS AND METABOLISM REPORTS, 2021, 28
[2] EFFECTS OF LONG-TERM MIGALASTAT TREATMENT ON RENAL FUNCTION BY BASELINE PROTEINURIA IN PATIENTS (PTS) WITH FABRY DISEASE
Schiffmann, Raphael
Bichet, Daniel
Germain, Dominique
Giugliani, Roberto
Hughes, Derralynn
Nicholls, Kathleen
Wilcox, William
Williams, Hadis
Yu, Julie
Castelli, Jeffrey
Skuban, Nina
Barth, Jay
NEPHROLOGY DIALYSIS TRANSPLANTATION, 2018, 33 : 347 - 348
[3] Long-term follow-up of renal function in patients treated with migalastat for Fabry disease (vol 28, 100786, 2021)
Bichet, Daniel G.
Torra, Roser
Wallace, Eric
Hughes, Derralynn
Giugliani, Roberto
Skuban, Nina
Krusinska, Eva
Feldt-Rasmussen, Ulla
Schiffmann, Raphael
Nicholls, Kathy
MOLECULAR GENETICS AND METABOLISM REPORTS, 2021, 29
[4] Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes
Hughes, Derralynn A.
Bichet, Daniel G.
Giugliani, Roberto
Hopkin, Robert J.
Krusinska, Eva
Nicholls, Kathleen
Olivotto, Iacopo
Feldt-Rasmussen, Ulla
Sakai, Norio
Skuban, Nina
Sunder-Plassmann, Gere
Torra, Roser
Wilcox, William R.
JOURNAL OF MEDICAL GENETICS, 2023, 60 (07) : 722 - 731
[5] Long term renal function in patients with Fabry disease
Politei, Juan
Heguilen, Ricardo
Cabrera, Gustavo
Bernasconi, Amelia
Durand, Consuelo
Joaquin, Frabasil
Beatriz Schenone, Andrea
MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S119 - S120
[6] Long-term enzyme replacement therapy for Fabry disease: efficacy and unmet needs in cardiac and renal outcomes
Ja Hye Kim
Beom Hee Lee
Ja Hyang Cho
Eungu Kang
Jin-Ho Choi
Gu-Hwan Kim
Han-Wook Yoo
Journal of Human Genetics, 2016, 61 : 923 - 929
[7] Long-term enzyme replacement therapy for Fabry disease: efficacy and unmet needs in cardiac and renal outcomes
Kim, Ja Hye
Lee, Beom Hee
Cho, Ja Hyang
Kang, Eungu
Choi, Jin-Ho
Kim, Gu-Hwan
Yoo, Han-Wook
JOURNAL OF HUMAN GENETICS, 2016, 61 (11) : 923 - 929
[8] Long-term impact of cardio-renal involvement on adverse outcomes in patients with Fabry disease
Siegenthaler, Martin
Huynh-Do, Uyen
Widmer, Urs
Battegay, Edouard
Krayenbuhl, Pierre-Alexandre
Nowak, Albina
SWISS MEDICAL WEEKLY, 2015, 145 : 2S - 3S
[9] Renal function predicts long-term outcome on enzyme replacement therapy in patients with Fabry disease
Lenders, Malte
Schmitz, Boris
Stypmann, Joerg
Duning, Thomas
Brand, Stefan-Martin
Kurschat, Christine
Brand, Eva
NEPHROLOGY DIALYSIS TRANSPLANTATION, 2017, 32 (12) : 2090 - 2097
[10] Evaluation of long-term renal outcomes in Fabry disease: A single centre analysis
Jovanovic, Ana
Chinnadurai, Rajkumar
Stepien, Karolina M.
Sharma, Reena
Kalra, Philip
Mannan, Fahmida
MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)

← 1 2 3 4 5 →