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Combined oxidative phosphorylation deficiency 47

MONDO:0033537

Also known as: COMBINED OXIDATIVE PHOSPHORYLATION DEFICIENCY 47, COXPD47

13 clinical trials for this condition and its sub-types, 0 tagged with Combined oxidative phosphorylation deficiency 47 itself.

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Part of

↑ Combined oxidative phosphorylation deficiency (0)

Including sub-types (13) Tagged with Combined oxidative phosphorylation deficiency 47 (0)

Trials to join now! 5 Not yet recruiting 1 Not yet finished but already full! 4 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