Hope for babies with rare brain disorder: new trial offers early rescue
NCT ID NCT04143295
First seen Jan 05, 2026 · Last updated May 15, 2026 · Updated 17 times
Summary
This study aims to help male infants with a rare genetic condition called MCT8 deficiency, which causes severe intellectual disability and movement problems. The trial enrolls babies with a confirmed gene mutation whose families choose not to end the pregnancy. The goal is to start treatment early to improve brain development and motor skills.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University of Miami, Miller School of Medicine
AVAILABLEMiami, Florida, 33136, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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