New hope for fabry patients: long-term drug shows safety promise
NCT ID NCT04049760
First seen Feb 05, 2026 · Last updated May 01, 2026 · Updated 16 times
Summary
This study looked at the long-term safety of a drug called migalastat in 16 people over age 12 with Fabry disease who have certain genetic changes. Fabry disease is a rare inherited condition that can cause pain, organ damage, and other serious problems. The goal was to see if taking migalastat over time is safe and how it affects kidney function and protein levels in urine.
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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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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
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Lysosomal & Rare Disorders Research & Treatment Center
Fairfax, Virginia, 22030, United States
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Royal Free London NHS Foundation Trust
London, United Kingdom
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The Emory Clinic
Atlanta, Georgia, 30322, United States
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University of Minnesota Masonic Children's Hospital and Clinics
Minneapolis, Minnesota, 55454, United States
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University of South Florida
Tampa, Florida, 33606, United States
Conditions
Explore the condition pages connected to this study.