LEIGH SYNDROME
Clinical trials for LEIGH SYNDROME explained in plain language.
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Last chance access: vatiquinone for mitochondrial disease patients
Disease control NO_LONGER_AVAILABLEThis program offered vatiquinone, an experimental liquid medication, to patients with inherited mitochondrial diseases like Leigh syndrome who had already completed a previous safety study. The goal was to continue treatment for those who might benefit, but enrollment is now clos…
Matched conditions: LEIGH SYNDROME
Sponsor: Medical University of South Carolina • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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Could a common drug help kids with rare leigh syndrome?
Disease control ENROLLING_BY_INVITATIONThis study tests the drug sirolimus (rapamycin) in 15 people aged 6 months to 55 years with genetically confirmed Leigh syndrome, a rare and serious mitochondrial disease. The goal is to see if the drug is safe and can help with symptoms like muscle weakness and developmental del…
Matched conditions: LEIGH SYNDROME
Phase: PHASE2 • Sponsor: Matthew Demczko • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC