A composite measurement concept for monitoring cardiac function in Fabry disease

Abstract Background Fabry disease (FD) is an X-linked, multisystemic, progressive lysosomal disorder caused by GLA variants resulting in alpha-galactosidase A deficiency. Although cardiovascular disease is the leading cause of death in people with FD, the progression of cardiac dysfunction remains p...

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Bibliographic Details
Main Authors: Pooja Nandi, Robert Ellis, Jennifer Hiros, Paul Howard, Biliana O. Veleva-Rotse
Format: Article
Language:English
Published: BMC 2025-07-01
Series:Orphanet Journal of Rare Diseases
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Online Access:https://doi.org/10.1186/s13023-025-03895-x
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