Mapping the health management journey of Fabry disease patients with cardiac involvement

Abstract excerpt

<title>Abstract</title> <p> <bold>Background</bold> Fabry disease is a rare X-linked genetic disorder that frequently involves multiple organ systems, with cardiac complications being the leading cause of death. Although enzyme replacement therapy can slow disease progression, patients continue to face significant physical and emotional challenges. Most research has focused on pathophysiology, leaving a gap in...

Topics

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• alpha-Galactosidase
• Child
• Enzyme Replacement Therapy
• Fabry Disease
• Mutation
• Phenotype
• Quality of Life
• Surveys and Questionnaires

Identifiers and source

Literature Corpus work

4ff494f9-f050-56f1-9aee-a0935b6d7ebb

DOI

10.21203/rs.3.rs-9189525/v1