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Clinical Trials for Leigh Disease

  • A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS)
  • Sirolimus for Leigh Syndrome
  • The KHENERGYC Study
  • A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
  • ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome
  • A Dose-escalating Clinical Trial With KH176
  • Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
  • Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
  • Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
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