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Multiple acyl-CoA dehydrogenase deficiency, severe neonatal type

MONDO:0018332

Also known as: MAD deficiency, severe neonatal type, MADD, severe neonatal type, glutaric aciduria type 2, severe neonatal type

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) Inherited lipid metabolism disorder (189) Hereditary disease (176) Cardiomyopathy (144) Hypertrophic cardiomyopathy (112) Inborn mitochondrial metabolism disorder (58)

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