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Leigh syndrome

MONDO:0009723

A progressive neurological disease defined by specific neuropathological features associating brainstem and basal ganglia lesions.

Also known as: LS, LSS, Leigh disease, Leigh syndrome, Leigh syndrome spectrum, Leigh's disease, infantile subacute necrotizing encephalopathy, Leigh syndrome due to mitochondrial Complex 1 deficiency

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

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Sub-types

Maternally-inherited Leigh syndrome (1) Congenital lactic acidosis, Saguenay-Lac-Saint-Jean type (0) Leigh syndrome with cardiomyopathy (0) Necrotizing encephalomyelopathy, subacute, of Leigh, adult (0)

Broader categories

Disease (618) Nervous system disorder (217) Metabolic disease (215) Hereditary disease (172) Peripheral nervous system disorder (107) Neuromuscular disease (98) Peripheral neuropathy (90) Inborn mitochondrial metabolism disorder (57) Inborn errors of metabolism (42) Mitochondrial disease (39)

Trials to join now! 24 Not yet recruiting 5 Not yet finished but already full! 8 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