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

Abstract 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 d...

Deskribapen osoa

Gorde:
Egile Nagusiak: Raphael Schiffmann, Daniel G. Bichet, Ana Jovanovic, Derralynn A. Hughes, Roberto Giugliani, Ulla Feldt-Rasmussen, Suma P. Shankar, Laura Barisoni, Robert B. Colvin, J. Charles Jennette, Fred Holdbrook, Andrew Mulberg, Jeffrey P. Castelli, Nina Skuban, Jay A. Barth, Kathleen Nicholls
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: BMC 2018-04-01
Saila:Orphanet Journal of Rare Diseases
Gaiak:
Amenable mutation
Diarrhea
Fabry disease
Gastrointestinal
Globotriaosylceramide
GSRS
Sarrera elektronikoa:http://link.springer.com/article/10.1186/s13023-018-0813-7
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