Could a common drug slow a rare brain disease?
NCT ID NCT06843811
First seen Apr 03, 2026 · Last updated May 18, 2026 · Updated 7 times
Summary
This study tests the drug sirolimus in 15 people with Leigh syndrome, a rare genetic disorder that damages the brain and muscles. The goal is to see if sirolimus is safe and can help manage symptoms. Participants must be between 6 months and 55 years old and have a confirmed genetic diagnosis.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146, United States
Conditions
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