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FASTKD2-related infantile mitochondrial encephalomyopathy

MONDO:0015632

13 clinical trials for this condition and its sub-types, 0 tagged with FASTKD2-related infantile mitochondrial encephalomyopathy itself.

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Where it sits in the disease tree

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Part of

↑ Mitochondrial oxidative phosphorylation disorder (58)

Including sub-types (13) Tagged with FASTKD2-related infantile mitochondrial encephalomyopathy (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