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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Dettagli Bibliografici
Pubblicato in:Orphanet J Rare Dis
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2018
Soggetti:
Research
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5923014/
https://ncbi.nlm.nih.gov/pubmed/29703262
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0813-7
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