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

21 clinical trials for this condition and its sub-types, 9 tagged with Leigh syndrome itself.

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Part of

↑ Hereditary peripheral neuropathy (479) ↑ Mitochondrial oxidative phosphorylation disorder (58)

Sub-types of Leigh syndrome

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

Including sub-types (21) Tagged with Leigh syndrome (9)

Trials to join now! 3 Not yet recruiting 1 Not yet finished but already full! 3 Completed 1 Terminated 1

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

Disease control Stopped early

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 2/3 • Sponsor: PTC Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 12:03 UTC

Leigh syndrome

Where it sits in the disease tree

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