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Mitochondrial DNA depletion syndrome, hepatocerebrorenal form

MONDO:0018197

Also known as: mtDNA depletion syndrome, hepatocerebrorenal form

51 clinical trials for this condition and its sub-types.

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Broader categories

Disease (618) Metabolic disease (215) Kidney disorder (202) Hereditary disease (172) Urinary system disorder (62) Inborn mitochondrial metabolism disorder (57) Inborn errors of metabolism (42) Mitochondrial disease (39) Human disease (14) Developmental defect during embryogenesis (7)

Trials to join now! 24 Not yet recruiting 6 Not yet finished but already full! 10 Completed 10 Terminated 1

New drug aims to tame hard-to-control seizures in rare mitochondrial disorders

Disease control Terminated

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

Phase: PHASE2, PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 12:03 UTC