Treatment needs and expectations for Fabry disease in France: development of a new Patient Needs Questionnaire

BACKGROUND: Fabry disease (FD) is a rare, X-linked, inherited lysosomal disease caused by absent or reduced α-galactosidase A activity. Due to the heterogeneity of disease presentation and progression, generic patient-reported outcome (PRO) tools do not provide accurate insight into patients’ daily...

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Detalles Bibliográficos
Autores principales: Noël, Esther, Dussol, Bertrand, Lacombe, Didier, Bedreddine, Najya, Fouilhoux, Alain, Ronco, Pierre, Genevaz, Delphine, Bekri, Soumeya, Hagège, Albert, Dupuis-Siméon, Frédérique, Derrien Ansquer, Valérie, Germain, Dominique P., Lidove, Olivier
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6894302/
https://www.ncbi.nlm.nih.gov/pubmed/31801581
http://dx.doi.org/10.1186/s13023-019-1254-7

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