Phase 2 Study of EPI-743 in Children With Pearson Syndrome

Inclusion Criteria: * Genetically confirmed diagnosis of Pearson syndrome * Age less than 18 * Availability of medical history for 12 months prior to enrollment * Abstention from use of CoQ10, vitamin E, lipoic acid and Idebenone 14 days prior to treatment with EPI-743 Exclusion Criteria: * Allergy to EPI-743, sesame oil or vitamin E * Clinical history of bleeding/ abnormal PT/PTT * Concurrent inborn errors of metabolism * Use of anticoagulant medications * Participation in any interventional study within 30 days of treatment * Use of erythropoietin 30 days prior to trial enrollment