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Mitochondrial myopathy with a defect in mitochondrial-protein transport

MONDO:0009638

Also known as: mitochondrial myopathy with a defect in mitochondrial-protein transport

18 clinical trials for this condition and its sub-types, 0 tagged with Mitochondrial myopathy with a defect in mitochondrial-protein transport itself.

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Where it sits in the disease tree

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Part of

↑ Inborn mitochondrial myopathy (45)

Including sub-types (18) Tagged with Mitochondrial myopathy with a defect in mitochondrial-protein transport (0)

Trials to join now! 10 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