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Article

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

Orphanet journal of rare diseases - 27 Apr 2018

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

Abstract excerpt

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...

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Identifiers

PMID: 29703262
DOI: 10.1186/s13023-018-0813-7
PMCID: PMC5923014

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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