Hope for fabry patients: new drug shows promise in Long-Term trial
NCT ID NCT03614234
First seen Jan 26, 2026 · Last updated Apr 28, 2026 · Updated 12 times
Summary
This study looks at the long-term safety and effectiveness of a drug called pegunigalsidase alfa for adults with Fabry disease, a rare genetic condition that can damage organs. Participants receive an infusion every four weeks and are monitored for side effects, kidney function, and heart health. The goal is to see if this treatment can help control the disease over time.
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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Azienda Ospedaliera Universitaria "Federico II"
Naples, Via Pansini, 80131, Italy
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Emory University School of Medicine
Atlanta, Georgia, 30307, United States
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Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
Prague, 128 08, Czechia
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Helse Bergen HF Haukeland Universitetssykehus
Bergen, 5021, Norway
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Infusion Associates
Grand Rapids, Michigan, 49525, United States
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Medical Endocrinology PE 2132, Rigshospitalet
Copenhagen, 2100, Denmark
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O & O Alpan
Fairfax, Virginia, 22030, United States
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Renal Disease Research Institute, LLC
Dallas, Texas, 75235, United States
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The Royal Free Hospital
London, United Kingdom
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UAB Medicine
Birmingham, Alabama, 35233, United States
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UZ Antwerpen
Edegem, 2650, Belgium
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University of Iowa Hospitals and Clinica
Iowa City, Iowa, 52242, United States
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University of Utah Hospitals & Clinics
Salt Lake City, Utah, 84112, United States
Conditions
Explore the condition pages connected to this study.