Pearson Syndrome Clinical Trial
Official title:
An Open-Label Phase 2 Safety and Efficacy Study of EPI-743 (VincerinoneTM) in Children With Pearson Syndrome
Treatment of Pediatric Subjects 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. ;
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