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Dystonia, childhood-onset, with optic atrophy and basal ganglia abnormalities

MONDO:0015003

Also known as: DYTOABG, dystonia, childhood-onset, with optic atrophy and basal ganglia abnormalities, dystonia, childhood-onset, with optic atrophy and basal ganglia abnormalities; DYTOABG, dystonia 29, childhood-onset

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

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Broader categories

Disease (618) Nervous system disorder (217) Metabolic disease (215) Hereditary disease (172) Inherited lipid metabolism disorder (165) Neurodegenerative disease (155) Central nervous system disorder (97) Movement disorder (89) Dystonic disorder (60) Inborn mitochondrial metabolism disorder (57)

Trials to join now! 36 Not yet recruiting 6 Not yet finished but already full! 9 Completed 23 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