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

If effective, treatment with EPI-743 should result in a significant reduction in occurrence
of sepsis, metabolic crisis, hepatic failure and transfusion dependence.

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