Gene therapy aims to fix fabry disease with one infusion
NCT ID NCT04046224
First seen Apr 23, 2026 · Last updated Apr 28, 2026 · Updated 1 time
Summary
This study tested a new gene therapy called ST-920 in 36 adults with Fabry disease. The treatment uses a harmless virus to deliver a working gene that helps the body produce an enzyme it is missing. The goal was to see if a single intravenous dose is safe and tolerable, and whether it can reduce harmful substances linked to the disease. Participants were followed for one year after the infusion.
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This is a summary of
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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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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Azienda Ospedaliero-Universitaria Careggi
Florence, Tuscany, 50134, Italy
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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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Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginia, 22030, United States
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M.A.G.I.C. Clinic Ltd.
Calgary, Alberta, T2E 7Z4, Canada
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Mt. Sinai School of Medicine
New York, New York, 10029, United States
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National Taiwan University Hospital
Taipei, Taiwan
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Queen Elizabeth Hospital
Birmingham, B15 2TH, United Kingdom
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Royal Free Hospital
London, United Kingdom
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The Royal Melbourne Hospital
Parkville, Victoria, 3050, Australia
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University Hospital of Würzburg
Würzburg, Germany
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University Medical Center Hamburg-Eppendorf
Hamburg, Germany
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University of California, Irvine
Irvine, California, 92697, United States
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University of Iowa Hospital and Clinics
Iowa City, Iowa, 52242, United States
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University of Minnesota Medical Center
Minneapolis, Minnesota, 55455, United States
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University of South Florida
Tampa, Florida, 33620, United States
Conditions
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