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Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial

BACKGROUND: Fabry disease is frequently characterized by gastrointestinal symptoms, including diarrhea. Migalastat is an orally-administered small molecule approved to treat the symptoms of Fabry disease in patients with amenable mutations. METHODS: We evaluated minimal clinically important differen...

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Detalles Bibliográficos
Autores principales:	Schiffmann, Raphael, Bichet, Daniel G., Jovanovic, Ana, Hughes, Derralynn A., Giugliani, Roberto, Feldt-Rasmussen, Ulla, Shankar, Suma P., Barisoni, Laura, Colvin, Robert B., Jennette, J. Charles, Holdbrook, Fred, Mulberg, Andrew, Castelli, Jeffrey P., Skuban, Nina, Barth, Jay A., Nicholls, Kathleen
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2018
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5923014/ https://www.ncbi.nlm.nih.gov/pubmed/29703262 http://dx.doi.org/10.1186/s13023-018-0813-7

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