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

ABSTRACT:

Objective

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.

Methods

A modified Delphi process was conducted to determine consensus on treatment decisions and monitoring of patients with Fabry disease receiving migalastat. The multidisciplinary panel comprised 14 expert physicians across nine specialties and two patients with Fabry

SUBMITTER: Bichet DG 

PROVIDER: S-EPMC10505750 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature