Encephalopathy due to defective mitochondrial and peroxisomal fission 2

MONDO:0014905

Also known as: EMPF2, Leigh-like basal ganglia disease-optic atrophy-peripheral neuropathy syndrome, MFF-associated encephalopathy due to peroxisomal and mitochondrial fission defect, encephalopathy due to defective mitochondrial and peroxisomal fission 2, encephalopathy due to defective mitochondrial and peroxisomal fission type 2

14 clinical trials for this condition and its sub-types, 0 tagged with Encephalopathy due to defective mitochondrial and peroxisomal fission 2 itself.

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Where it sits in the disease tree

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

Including sub-types (14) Tagged with Encephalopathy due to defective mitochondrial and peroxisomal fission 2 (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 due to defective mitochondrial and peroxisomal fission 2

Where it sits in the disease tree

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