https://scholars.lib.ntu.edu.tw/handle/123456789/636690
Title: | Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study | Authors: | Bichet, Daniel G Hopkin, Robert J Aguiar, Patrício Allam, Sridhar R YIN-HSIU CHIEN Giugliani, Roberto Kallish, Staci Kineen, Sabina Lidove, Olivier Niu, Dau-Ming Olivotto, Iacopo Politei, Juan Rakoski, Paul Torra, Roser Tøndel, Camilla Hughes, Derralynn A |
Keywords: | alpha-galactosidase A; amenability; chaperone therapy; globotriaosylsphingosine; patient journey; treatment decisions | Issue Date: | 2023 | Journal Volume: | 10 | Source: | Frontiers in medicine | 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. |
URI: | https://scholars.lib.ntu.edu.tw/handle/123456789/636690 | ISSN: | 2296-858X | DOI: | 10.3389/fmed.2023.1220637 |
Appears in Collections: | 醫學系 |
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