In Vitro and In Vivo Amenability to Migalastat in Fabry Disease
Author:
Publisher
Elsevier BV
Subject
Genetics,Molecular Biology,Molecular Medicine
Reference46 articles.
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2. Investigating Fabry disease - some lessons learned;Rare Disease and Orphan Drugs Journal;2024-01-23
3. A review and recommendations for oral chaperone therapy in adult patients with Fabry disease;Orphanet Journal of Rare Diseases;2024-01-18
4. Fabry disease biomarkers in patients switched from enzyme-replacement therapy to migalastat oral chaperone therapy;Bioanalysis;2023-12
5. Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study;Frontiers in Medicine;2023-09-01
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