New shot aims to strengthen muscles in rare genetic disease
NCT ID NCT07531251
First seen Apr 24, 2026 · Last updated May 05, 2026 · Updated 4 times
Summary
This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare genetic condition that causes muscle weakness and heart problems. The goal is to see if the drug improves muscle strength and mobility over 72 weeks compared to a placebo. Participants will receive either the drug or a placebo injection daily, and their ability to walk, stand up, and move will be measured.
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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
Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman Building
Bristol, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Trial Not Offered in the U.S
Needham, Massachusetts, 02494, United States
Conditions
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