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

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

ObjectiveFabry disease is a progressive disorder caused by deficiency of the α-galactosidase A enzyme (α-Gal A), leading to multisystemic organ damage with heterogenous clinical presentation. The addition of the oral chaperone therapy migalastat to the available treatment options for Fabry disease i...

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Bibliographic Details
Main Authors: 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, Iacopo Olivotto, Juan Politei, Paul Rakoski, Roser Torra, Camilla Tøndel, Derralynn A. Hughes
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-09-01
Series:Frontiers in Medicine
Subjects:
chaperone therapy
alpha-galactosidase A
globotriaosylsphingosine
amenability
treatment decisions
patient journey
Online Access:https://www.frontiersin.org/articles/10.3389/fmed.2023.1220637/full

Internet

https://www.frontiersin.org/articles/10.3389/fmed.2023.1220637/full

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