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

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

Frontiers in medicine（2023）

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摘要

These recommendations build on previously published guidelines to highlight the importance of holistic, multidisciplinary monitoring for patients with Fabry disease receiving migalastat, in addition to shared decision-making regarding treatments and monitoring throughout the patient journey. GRAPHICAL ABSTRACT.

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关键词

chaperone therapy, alpha-galactosidase A, globotriaosylsphingosine, amenability, treatment decisions, patient journey

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