Last Updated: 09/26/2026
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Found 24 publications
Porcine kidney xenotransplantation as a bridge to allotransplantation: a first-in-human study.
Journal: Lancet (London, England)
Published: April 16, 2026
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia.
Journal: Human gene therapy
Published: May 11, 2023
FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument.
Journal: Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation
Published: April 12, 2021
Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study.
Journal: Molecular genetics and metabolism
Published: March 27, 2020
Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study).
Journal: Orphanet journal of rare diseases
Published: February 20, 2020
Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study.
Journal: Orphanet journal of rare diseases
Published: February 20, 2020
Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report.
Journal: Case reports in obstetrics and gynecology
Published: August 23, 2019
Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study.
Journal: Clinical and experimental nephrology
Published: July 19, 2019
Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study.
Journal: American journal of medical genetics. Part A
Published: October 07, 2018
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.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: August 09, 2018
Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial.
Journal: Orphanet journal of rare diseases
Published: November 29, 2017
Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment.
Journal: Journal of medical genetics
Published: May 19, 2017
Last Updated: 09/26/2026