New hope for fabry patients: Long-Term drug safety confirmed
NCT ID NCT04049760
First seen Feb 05, 2026 · Last updated Jun 13, 2026 · Updated 24 times
Summary
This study looked at the long-term safety of a drug called migalastat in 16 people over age 12 with Fabry disease, a rare genetic disorder. Participants had already completed an earlier study and continued taking migalastat to see if it was safe and how it affected kidney function. The goal was to manage the disease over time, not to cure it.
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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.