Browsing by Subject "Migalastat"
Now showing items 1-3 of 3
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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
(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 ... -
Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
(2021) [Journal article]The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)–naive and ERTexperienced patients with Fabry disease is not well defined. An integrated posthoc analysis of the phase 3 clinical ... -
Migalastat improves diarrhea in patients with Fabry disease : clinical-biomarker correlations from the phase 3 FACETS trial
(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 ...