Encephalopathy, lethal, due to defective mitochondrial peroxisomal fission 1
MONDO:0013726Also known as: DNM1L-associated encephalopathy due to peroxisomal and mitochondrial fission defect, encephalopathy, lethal, due to defective mitochondrial peroxisomal fission 1, lethal encephalopathy due to mitochondrial and peroxisomal fission defect, EMPF, EMPF1, encephalopathy due to defective mitochondrial and peroxisomal fission 1, encephalopathy, lethal, due to defective mitochondrial and peroxisomal fission
14 clinical trials for this condition and its sub-types, 0 tagged with Encephalopathy, lethal, due to defective mitochondrial peroxisomal fission 1 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
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New diagnostic strategy aims to end diagnostic odyssey for rare diseases
Diagnosis CompletedThis study tested a new approach to diagnose peroxisomal disorders, a group of rare genetic diseases. The strategy uses advanced metabolic and genetic tests to find the cause faster in people with suspicious symptoms or lab results. Researchers included 8 participants from four h…
Sponsor: University Hospital, Lille • Aim: Diagnosis
Last updated Jun 27, 2026 08:02 UTC