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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

21 clinical trials for this condition and its sub-types, 0 tagged with Multiple acyl-CoA dehydrogenase deficiency, severe neonatal type itself.

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↑ Multiple acyl-CoA dehydrogenase deficiency (1)

Including sub-types (21) Tagged with Multiple acyl-CoA dehydrogenase deficiency, severe neonatal type (0)

Trials to join now! 10 Not yet recruiting 1 Not yet finished but already full! 7 Completed 2 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