Cure My Disease

Don't miss your cure!

Log in / Sign up

Recessive mitochondrial ataxia syndrome

MONDO:0019791

Also known as: MIRAS

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

Follow this condition — get notified about new trials

Broader categories

Disease (618) Nervous system disorder (217) Metabolic disease (215) Hereditary disease (172) Neurodegenerative disease (155) Brain disorder (110) Central nervous system disorder (97) Movement disorder (89) Inborn mitochondrial metabolism disorder (57) Inborn errors of metabolism (42)

Trials to join now! 34 Not yet recruiting 7 Not yet finished but already full! 11 Completed 16 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