A review and recommendations for oral chaperone therapy in adult patients with Fabry disease.
Nowicki M, Bazan-Socha S, Błażejewska-Hyżorek B, Kłopotowski MM, Komar M, Kusztal MA, Liberek T, Małyszko J, Mizia-Stec K, Oko-Sarnowska Z, Pawlaczyk K, Podolec P, Sławek J; Polish Fabry Disease Collaborative Group.
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Orphanet J Rare Dis. 2024 Jan 18;19(1):16. doi: 10.1186/s13023-024-03028-w.
Orphanet J Rare Dis. 2024.
PMID: 38238782
Free PMC article.
Review.