Stephen Waldek
Sunderland, ENG, United Kingdom
Last Updated: 09/26/2026
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Found 21 publications
Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study.
Journal: Orphanet journal of rare diseases
Published: November 12, 2025
A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies.
Journal: Journal of inherited metabolic disease
Published: February 23, 2024
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.
Journal: Journal of medical genetics
Published: June 02, 2023
Fabry disease revisited: Management and treatment recommendations for adult patients.
Journal: Molecular genetics and metabolism
Published: December 18, 2017
Phenotypic characteristics of the p.Asn215Ser (p.N215S) GLA mutation in male and female patients with Fabry disease: A multicenter Fabry Registry study.
Journal: Molecular genetics & genomic medicine
Published: October 15, 2017
Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.
Journal: The New England journal of medicine
Published: August 11, 2016
Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry.
Journal: Molecular genetics and metabolism
Published: April 26, 2016
Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry.
Journal: Journal of medical genetics
Published: August 26, 2015
It is time to review how unlicensed medicines are used.
Journal: European journal of clinical pharmacology
Published: February 10, 2015
Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease.
Journal: Journal of medical genetics
Published: September 29, 2014
Chronic kidney disease and an uncertain diagnosis of Fabry disease: approach to a correct diagnosis.
Chronic kidney disease and an uncertain diagnosis of Fabry disease: approach to a correct diagnosis.
Journal: Molecular genetics and metabolism
Published: July 03, 2014
Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study.
Journal: Journal of inherited metabolic disease
Published: December 18, 2013
Last Updated: 09/26/2026