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FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument

PURPOSE: Fabry disease is a rare multisystemic disorder caused by functional deficiency of the lysosomal enzyme alpha-galactosidase A. Gastrointestinal (GI) signs and symptoms are among the earliest clinical manifestations in patients with Fabry disease but are often nonspecific, misdiagnosed, and u...

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Detalles Bibliográficos
Autores principales: Shields, Alan L., Lamoureux, Roger E., Taylor, Fiona, Barth, Jay A., Mulberg, Andrew E., Kessler, Vivian, Skuban, Nina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8481178/
https://www.ncbi.nlm.nih.gov/pubmed/33914257
http://dx.doi.org/10.1007/s11136-021-02847-9