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Myopathy, lactic acidosis, and sideroblastic anemia 3

MONDO:0010782

Also known as: myopathy, lactic acidosis, and sideroblastic anaemia type 3, myopathy, lactic acidosis, and sideroblastic anemia 3, myopathy, lactic acidosis, and sideroblastic anemia type 3, MLASA3

25 clinical trials for this condition and its sub-types.

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Broader categories

Disease (680) Anemia (233) Metabolic disease (233) Musculoskeletal system disorder (207) Hereditary disease (176) Hematologic disorder (170) Inborn mitochondrial metabolism disorder (58) Muscle tissue disorder (56) Inborn errors of metabolism (45) Mitochondrial disease (40)

Trials to join now! 13 Not yet recruiting 2 Not yet finished but already full! 5 Completed 4 Terminated 1

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

Disease control Terminated

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: PHASE2, PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 12:03 UTC