New hope for fabry patients: Long-Term enzyme therapy shows safety
NCT ID NCT03566017
First seen Apr 23, 2026 · Last updated Apr 30, 2026 · Updated 1 time
Summary
This study looked at the long-term safety of a drug called pegunigalsidase alfa in adults with Fabry disease, a rare genetic disorder. 97 people who had already completed earlier studies received the drug every two weeks. The main goal was to check for side effects over time, and no new safety concerns were found.
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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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Academisch Medisch Centrum
Amsterdam, 1105 AZ, Netherlands
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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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Capital District Health Authority
Halifax, Nova Scotia, B3H 1V8, Canada
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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General Hospital Slovenj Gradec
Slovenj Gradec, 2380, Slovenia
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Haukeland University Hospital Klinisk Forskningspost
Bergen, 5021, Norway
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Hospital Raymond-Poincaré
Garches, 92380, France
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Hospital de Dia Quiron Zaragoza
Zaragoza, 50012, Spain
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Infusion Associates
Grand Rapids, Michigan, 49525, United States
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Institute of Metabolic Disease
Dallas, Texas, 75246, United States
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Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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O+O Alpan LLC
Fairfax, Virginia, 22030, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Renal Disease Research Institute, LLC - Dallas
Dallas, Texas, 75235, United States
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Royal Melbourne Hospital
Parkville, Victoria, 3050, Australia
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Salford Royal
Salford, Greater Manchester, M6 8HD, United Kingdom
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Semmelweis Egyetem
Budapest, 1083, Hungary
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The Royal Free Hospital
London, NW3 2QG, United Kingdom
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Turku University Central Hospital
Turku, FI-20521, Finland
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UAB Medicine
Birmingham, Alabama, 35233, United States
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University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
Edgbaston, Birmingham, B152TH, United Kingdom
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University of California Irvine Center
Orange, California, 92868, United States
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University of California San Diego
La Jolla, California, 92037, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
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University of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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University of Utah Hospitals & Clinics
Salt Lake City, Utah, 84108, United States
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Vseobecna fakultni nemocnice v Praze
Prague, Czech Republic, 12808, Czechia
Conditions
Explore the condition pages connected to this study.