Encephalopathy, lethal, due to defective mitochondrial peroxisomal fission 1

MONDO:0013726

Also 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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↑ Mendelian encephalopathy (1) ↑ Encephalopathy due to mitochondrial and peroxisomal fission defect (0)

Including sub-types (14) Tagged with Encephalopathy, lethal, due to defective mitochondrial peroxisomal fission 1 (0)

Trials to join now! 5 Not yet recruiting 1 Not yet finished but already full! 4 Completed 3 Terminated 1

New drug aims to tame hard-to-control seizures in rare mitochondrial disorders

Disease control Stopped early

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

Phase 2/3 • Sponsor: PTC Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 12:03 UTC

Encephalopathy, lethal, due to defective mitochondrial peroxisomal fission 1

Where it sits in the disease tree

New drug aims to tame hard-to-control seizures in rare mitochondrial disorders