Browsing Biological Sciences by Author "Jennette, J. Charles"
Now showing items 1-2 of 2
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Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study
Germain, Dominique P.; Nicholls, Kathy; Giugliani, Roberto; Bichet, Daniel G.; Hughes, Derralynn A.; Barisoni, Laura; Colvin, Robert B.; Jennette, J. Charles; Skuban, Nina; Castelli, Jeffrey P.; Benjamin, Elfrida R.; Barth, Jay; Viereck, Christopher (2019) [Journal article]Purpose: Outcomes in patients with Fabry disease receiving migalastat during the phase 3 FACETS trial (NCT00925301) were evaluated by phenotype. Methods: Data were evaluated in two subgroups of patients with migalastat-amenable ... -
Migalastat improves diarrhea in patients with Fabry disease : clinical-biomarker correlations from the phase 3 FACETS trial
Schiffmann, Raphael; Bichet, Daniel G.; Jovanovic, Ana; Hughes, Derralynn A.; Giugliani, Roberto; Feldt-Rasmussen, Ulla; Shankar, S. P.; Barisoni, Laura; Colvin, Robert B.; Jennette, J. Charles; Holdbrook, Fred; Mulberg, Andrew E.; Castelli, Jeffrey P.; Skuban, Nina; Barth, Jay; Nicholls, Kathleen M. (2018) [Journal article]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 ...