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5 articles related to "Migalastat"

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Articles

Case Report

Published on 06 Jun 2024

Late-onset renal variant Fabry disease with R112H mutation and mild increase in plasma globotriaosylsphingosine: a case report

in Nephrology

Keiko Tanaka
Hitoshi Sugiyama
Hiroshi Morinaga
Akifumi Onishi
Katsuyuki Tanabe
Haruhito A. Uchida
Hiroki Maruyama
Jun Wada

Frontiers in Medicine

doi 10.3389/fmed.2024.1383309

2,857 views
3 citations

Original Research

Published on 01 Sep 2023

Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study

in Precision Medicine

Daniel G. Bichet
Robert J. Hopkin
Patrício Aguiar
Sridhar R. Allam
Yin-Hsiu Chien
Roberto Giugliani
Staci Kallish
Sabina Kineen
Olivier Lidove
Dau-Ming Niu

Frontiers in Medicine

doi 10.3389/fmed.2023.1220637

14,309 views
14 citations

Original Research

Published on 02 Nov 2023

Impact of enzyme replacement therapy and migalastat on left atrial strain and cardiomyopathy in patients with Fabry disease

in Cardiovascular Genetics and Systems Medicine

Christian Pogoda
Stefan-Martin Brand
Thomas Duning
Antje Schmidt-Pogoda
Jürgen Sindermann
Malte Lenders
Eva Brand

Frontiers in Cardiovascular Medicine

doi 10.3389/fcvm.2023.1223635

3,877 views
7 citations

Review

Published on 07 Jul 2017

Pharmaceutical Chaperones and Proteostasis Regulators in the Therapy of Lysosomal Storage Disorders: Current Perspective and Future Promises

in Experimental Pharmacology and Drug Discovery

Fedah E. Mohamed
Lihadh Al-Gazali
Fatma Al-Jasmi
Bassam R. Ali

Frontiers in Pharmacology

doi 10.3389/fphar.2017.00448

14,463 views
62 citations

Case Report

Published on 14 Jun 2023

Discordant renal progression of Fabry disease in male monozygotic twins: a case report

in Genetics of Common and Rare Diseases

Do-Yun Lee
Jun-Yeong Jeong
Seung-Eun Lee
Jae-Hun Lee
Ju-Young Moon
Su Woong Jung
Sang-Ho Lee
Yang Gyun Kim

Frontiers in Genetics

doi 10.3389/fgene.2023.1150822

3,187 views
2 citations