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Renal tubulopathy-encephalopathy-liver failure syndrome

MONDO:0016811

Renal tubulopathy - encephalopathy - liver failure describes a spectrum of phenotypes with manifestations similar but milder than those seen in Gracile syndrome and that can be associated with encephalopathy and psychiatric disorders.

13 clinical trials for this condition and its sub-types, 0 tagged with Renal tubulopathy-encephalopathy-liver failure syndrome itself.

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Where it sits in the disease tree

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Part of

↑ Mitochondrial complex III deficiency nuclear type 1 (0)

Including sub-types (13) Tagged with Renal tubulopathy-encephalopathy-liver failure syndrome (0)

Trials to join now! 5 Not yet recruiting 1 Not yet finished but already full! 4 Completed 2 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