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Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study
Journal
Frontiers in medicine
Journal Volume
10
Date Issued
2023
Author(s)
Bichet, Daniel G
Hopkin, Robert J
Aguiar, Patrício
Allam, Sridhar R
Giugliani, Roberto
Kallish, Staci
Kineen, Sabina
Lidove, Olivier
Niu, Dau-Ming
Olivotto, Iacopo
Politei, Juan
Rakoski, Paul
Torra, Roser
Tøndel, Camilla
Hughes, Derralynn A
Abstract
Fabry 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 is not yet universally reflected in all treatment guidelines. These consensus recommendations are intended to provide guidance for the treatment and monitoring of patients with Fabry disease receiving migalastat.
Subjects
alpha-galactosidase A; amenability; chaperone therapy; globotriaosylsphingosine; patient journey; treatment decisions
Type
journal article