Study shows rare disease patients need lifelong medication
NCT ID NCT05579327
Summary
This study tested what happens when males with MCT8 deficiency (a rare genetic disorder affecting thyroid hormones) stop taking their medication tiratricol. Researchers gave 20 participants either the real medication or a placebo for 30 days to see if stopping treatment caused dangerous hormone level increases. The results confirm that patients need to continue taking this medication long-term to control their condition.
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Contacts and locations
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Locations
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Addenbrooke's Hospital
Cambridge, United Kingdom
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Erasmus MC
Rotterdam, 3015 GD, Netherlands
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Rare Disease Research, LLC
Kissimmee, Florida, 34746, United States
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Rare Disease Research, LLC
Atlanta, Georgia, 30329, United States
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Rare Disease Research, LLC
Hillsborough, North Carolina, 27278, United States
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SSM Health Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104, United States
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Tranquil Clinical and Research Consulting Services
Webster, Texas, 77598, United States
Conditions
Explore the condition pages connected to this study.