Two-Year trial tests Drug's power to control rare genetic disease
NCT ID NCT04049760
Summary
This study followed 16 patients over 12 years old with a specific type of Fabry disease for two years. The goal was to see if the drug migalastat was safe and effective for long-term use in controlling this rare genetic disorder. Researchers tracked side effects, kidney and heart health, pain levels, and overall quality of life.
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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
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