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2 studies found for:    "pyruvate carboxylase deficiency" OR "Pyruvate Metabolism, Inborn Errors"

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Rank Status Study
1 Recruiting Tissue Sample Study for Mitochondrial Disorders
Conditions: Mitochondrial Disorders;   Mitochondrial Disease;   Melas;   Kearns Sayer;   NARP;   MNGIE;   LHON;   Mitochondrial Depletion Syndrome;   Leigh's Disease
Intervention:
2 Recruiting Safety and Efficacy Study of EPI-743 in Children With Genetically Confirmed Leigh Syndrome
Condition: Leigh Syndrome
Interventions: Drug: Placebo;   Drug: EPI-743 15 mg/kg;   Drug: EPI-743 5 mg/kg
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† Indicates status has not been verified in more than two years