Records containing: Leigh Syndrome
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-03-31A Pilot Phase 2 Study With Long-Term Extension to Assess the Safety and Efficacy of Sirolimus in Patients With Leigh Syndrome Caused by Genetically-Confirmed Mitochondrial Respiratory Chain Deficiency
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CLINICAL_TRIALS study record update · 2026-03-25Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy
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CLINICAL_TRIALS study record update · 2025-07-11A Phase 2a Partially Randomized, Placebo-controlled, Sequential Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum
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CLINICAL_TRIALS study record update · 2025-06-05Global Mitochondrial Registry to Define Natural History and Outcome Measures to Achieve Definite Trial Readiness for Mitochondrial Disorders
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CLINICAL_TRIALS study record update · 2025-05-29Coordination of Rare Diseases at Sanford
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CLINICAL_TRIALS study record update · 2024-10-23Long-Term Safety and Efficacy Evaluation of Vatiquinone (EPI-743) in Children With Leigh Syndrome
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CLINICAL_TRIALS study record update · 2023-12-01The International Registry for Leigh Syndrome
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CLINICAL_TRIALS study record update · 2022-05-20A Natural History Study of Surfeit Locus Protein 1 (SURF1)-Associated Leigh Syndrome
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CLINICAL_TRIALS study record update · 2022-02-01A Phase 2a, Open-label Study to Evaluate the Safety, Tolerability, and Clinical Activity of ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome
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CLINICAL_TRIALS study record update · 2020-11-12A Phase I, Randomized, Double Blind, Placebo-controlled, Dose-escalating Clinical Trial With KH176
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CLINICAL_TRIALS study record update · 2020-08-31A Phase 2B Randomized, Placebo Controlled, Double Blind Clinical Trial of EPI-743 in Children With Leigh Syndrome