Leigh Syndrome
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism
The NIH MINI study is currently recruiting to study metabolism, infection, and immunity in people with inborn errors of metabolism.
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CLINICAL_TRIALS research · 2026-03-31Clinical Trial Update: Sirolimus for Leigh Syndrome [Phase 2]
A Phase 2 clinical trial is investigating sirolimus for Leigh syndrome, with enrollment currently listed as enrolling by invitation.
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CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy [Phase 2, Phase 3]
A Phase 2/3 study evaluating Vatiquinone for mitochondrial disease in participants with refractory epilepsy has been terminated, and results…
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CLINICAL_TRIALS research · 2026-01-07Clinical Trial Update: Tissue Sample Study for Mitochondrial Disorders
Columbia University is enrolling by invitation in a tissue-sample study for mitochondrial disorders, including MELAS, Kearns-Sayre syndrome,…
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CLINICAL_TRIALS research · 2025-07-11Clinical Trial Update: A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS) [Phase 2]
A Phase 2 dose-ranging clinical trial of TTI-0102 in adults and children with Leigh syndrome spectrum is listed as not yet recruiting.
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CLINICAL_TRIALS research · 2025-06-05Clinical Trial Update: Global Registry and Natural History Study for Mitochondrial Disorders
LMU Klinikum is conducting a global registry and natural history study to collect data on mitochondrial disorders, and the study is currentl…
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CLINICAL_TRIALS research · 2025-05-13Clinical Trial Update: Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
Clinical trial investigating repurposed drugs for Leigh syndrome using induced pluripotent stem cell derived neural cells; currently recruit…
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CLINICAL_TRIALS trial_result · 2025-01-10Clinical Trial Update: The KHENERGYC Study [Phase 2]
Phase 2 KHENERGYC study for mitochondrial diseases is currently suspended.
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CLINICAL_TRIALS trial_result · 2024-10-23Clinical Trial Update: Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome [Phase 2]
This document summarizes the Phase 2 long-term safety and efficacy results of EPI-743 in children with Leigh syndrome.
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CLINICAL_TRIALS trial_result · 2020-11-12Clinical Trial Update: A Dose-escalating Clinical Trial With KH176 [Phase 1]
Phase 1 dose-escalation trial of KH176 in mitochondrial diseases completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-08-31Clinical Trial Update: Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome [Phase 2]
Phase 2 clinical trial assessing safety and efficacy of EPI-743 in children with Leigh Syndrome has been completed.
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CLINICAL_TRIALS trial_result · 2017-11-13Clinical Trial Update: Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease [Phase 2]
Phase 2 open-label, dose-escalation study of RP103 to assess safety, tolerability, and efficacy in inherited mitochondrial diseases, includi…