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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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Detalles Bibliográficos
Autores principales: Hamed, Alaa, DasMahapatra, Pronabesh, Lyn, Nicole, Gwaltney, Chad, Iaconangelo, Charlie, Serrano, Daniel, Modur, Vijay, Politei, Juan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8639795/
https://www.ncbi.nlm.nih.gov/pubmed/34900595
http://dx.doi.org/10.1016/j.ymgmr.2021.100824