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Cardiac lipidosis, familial

MONDO:0008911

Also known as: cardiac lipidosis, familial

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

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Broader categories

Cardiovascular disorder (1051) Disease (680) Heart disorder (300) Metabolic disease (233) Musculoskeletal system disorder (207) Hereditary disease (176) Cardiomyopathy (144) Dilated cardiomyopathy (72) Inborn mitochondrial metabolism disorder (58) Muscle tissue disorder (56)

Trials to join now! 10 Not yet recruiting 1 Not yet finished but already full! 6 Completed 2 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