Myopathy, lactic acidosis, and sideroblastic anemia 2

MONDO:0013307

Any mitochondrial myopathy and sideroblastic anemia in which the cause of the disease is a mutation in the YARS2 gene.

Also known as: YARS2 mitochondrial myopathy and sideroblastic anaemia, YARS2 mitochondrial myopathy and sideroblastic anemia, mitochondrial myopathy and sideroblastic anaemia caused by mutation in YARS2, mitochondrial myopathy and sideroblastic anemia caused by mutation in YARS2, myopathy, lactic acidosis, and sideroblastic Anaemia type 2, myopathy, lactic acidosis, and sideroblastic Anemia type 2, myopathy, lactic acidosis, and sideroblastic anemia 2, MLASA2

27 clinical trials for this condition and its sub-types, 0 tagged with Myopathy, lactic acidosis, and sideroblastic anemia 2 itself.

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↑ Myopathy, lactic acidosis, and sideroblastic anemia (3)

Including sub-types (27) Tagged with Myopathy, lactic acidosis, and sideroblastic anemia 2 (0)

Trials to join now! 14 Not yet recruiting 3 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 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

Myopathy, lactic acidosis, and sideroblastic anemia 2

Where it sits in the disease tree

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