Inborn disorder of energy metabolism
MONDO:0019243An inherited metabolic disease that is has its basis in the disruption of generation of precursor metabolites and energy.
Also known as: inborn error of generation of precursor metabolites and energy, inborn generation of precursor metabolites and energy disorder, rare inborn error of generation of precursor metabolites and energy, disorder of energy metabolism
241 clinical trials for this condition and its sub-types.
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Broader categories
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New drug aims to tame hard-to-control seizures in rare mitochondrial disorders
Disease control TerminatedThis 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: PHASE2, PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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Pompe disease drug trial halted after just 3 patients
Disease control TerminatedThis study tested an oral drug called duvoglustat in adults with Pompe disease, a rare genetic disorder that causes muscle weakness. Only 3 people took part, and the study was stopped early. Researchers looked at safety and whether the drug could improve walking distance and othe…
Phase: PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 26, 2026 16:43 UTC