Mitochondrial DNA depletion syndrome 14B (cardioencephalomyopathic type)
MONDO:0014820Also known as: MTDPS14, OPA1 mitochondrial DNA depletion syndrome, mitochondrial DNA depletion syndrome 14 (cardioencephalomyopathic type), mitochondrial DNA depletion syndrome 14 (cardioencephalomyopathic type); MTDPS14, mitochondrial DNA depletion syndrome 14 (encephalocardiomyopathic type), mitochondrial DNA depletion syndrome caused by mutation in OPA1
13 clinical trials for this condition and its sub-types, 0 tagged with Mitochondrial DNA depletion syndrome 14B (cardioencephalomyopathic type) itself.
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New drug shows promise for rare energy disorder
Disease control CompletedThis study tested a new drug called OMT-28 in 28 people with primary mitochondrial disease, a condition that affects how cells produce energy. Participants took the drug once daily for 6 months, and researchers measured safety, blood markers of inflammation, and symptoms like fat…
Phase 2 • Sponsor: Omeicos Therapeutics GmbH • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Long-Term safety of mitochondrial drug confirmed in 101 patients
Disease control CompletedThis study looked at the safety of vatiquinone in 101 people with inherited mitochondrial disease who had already taken the drug in a previous study or treatment plan. The goal was to track any side effects until the drug became commercially available or the program ended. Partic…
Phase 3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC