Browsing Department of Clinical Science by Journals "Frontiers of Medicine"
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Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study
(Journal article; Peer reviewed, 2023-09-01)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 ...