Journal article
Migalastat improves diarrhea in patients with Fabry disease: Clinical-biomarker correlations from the phase 3 FACETS trial
R Schiffmann, DG Bichet, A Jovanovic, DA Hughes, R Giugliani, U Feldt-Rasmussen, SP Shankar, L Barisoni, RB Colvin, JC Jennette, F Holdbrook, A Mulberg, JP Castelli, N Skuban, JA Barth, K Nicholls
Orphanet Journal of Rare Diseases | BMC | Published : 2018
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 differences (MCID) in diarrhea based on the corresponding domain of the patient-reported Gastrointestinal Symptom Rating Scale (GSRS) in patients with Fabry disease and amenable mutations (N = 50) treated with migalastat 150 mg every other day or placebo during the phase 3 FACETS trial (NCT00925301). Results: After 6 months, significantly more patients receiving migalastat versus placebo experienced impro..
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Funding Acknowledgements
This study was funded by Amicus Therapeutics, Inc., Cranbury, NJ.