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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Where it sits in the disease tree

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Part of

↑ Hereditary peripheral neuropathy (478) ↑ 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

Long-Term safety of mitochondrial drug confirmed in 101 patients

Disease control Completed

This study looked at the safety of vatiquinone in 101 people with inherited mitochondrial disease who had already taken the drug in a previous study or treatment plan. The goal was to track any side effects until the drug became commercially available or the program ended. Partic…

Phase 3 • Sponsor: PTC Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 08:05 UTC

Leigh syndrome

Where it sits in the disease tree

Long-Term safety of mitochondrial drug confirmed in 101 patients