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Mitochondrial membrane transport disorder

MONDO:0016800

13 clinical trials for this condition and its sub-types, 0 tagged with Mitochondrial membrane transport disorder itself.

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Part of

↑ Inborn mitochondrial metabolism disorder (127)

Sub-types of Mitochondrial membrane transport disorder

  • Mitochondrial protein import disorder 0 trials
  • Mitochondrial substrate carrier disorder 0 trials
    5 sub-types
    • Sengers syndrome 0 trials
    • Cardiomyopathy-hypotonia-lactic acidosis syndrome 0 trials
    • Combined oxidative phosphorylation deficiency 28 0 trials
    • Developmental and epileptic encephalopathy, 39 0 trials
    • Sideroblastic anemia 3 0 trials
Including sub-types (13) Tagged with Mitochondrial membrane transport disorder (0)
Trials to join now! 5 Not yet recruiting 1 Not yet finished but already full! 4 Completed 2 Terminated 1
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  • 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

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