Fabry disease patient-reported outcome (FD-PRO) demonstrates robust measurement properties for assessing symptom severity in Fabry disease
Background: Fabry disease (FD) is a rare, genetic disease, that if untreated, progresses to irreversible and life-threatening renal, cardiac, and cerebrovascular events. FD symptoms impact daily functioning and quality of life, but no disease-specific measure of these symptoms has been psychometrica...
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Autores principales: | Alaa Hamed, Pronabesh DasMahapatra, Nicole Lyn, Chad Gwaltney, Charlie Iaconangelo, Daniel Serrano, Vijay Modur, Juan Politei |
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Formato: | article |
Lenguaje: | EN |
Publicado: |
Elsevier
2021
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Materias: | |
Acceso en línea: | https://doaj.org/article/d3371297b9c04aab9730b92e45a82f01 |
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