New hope for devastating childhood disease in drug trial
NCT ID NCT06843811
Summary
This study is testing whether a drug called sirolimus is safe and can help control symptoms in people with Leigh syndrome, a severe genetic disorder that affects the brain and muscles. About 15 participants, from infants to adults, will take the drug daily for up to two years. Researchers will measure changes in movement, strength, fatigue, and overall function to see if the treatment helps.
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Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146, United States
Conditions
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