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First-in-Human Mitochondrial Augmentation of Hematopoietic Stem Cells in Pearson Syndrome

Minovia conducted a compassionate use program at Sheba Medical Center, Tel Aviv, Israel, through which three children with Pearson syndrome and one patient with Kearns-Sayre syndrome were treated. Results were presented at the annual meeting of the American Society of Hematology (ASH) in San Diego, December 2018. This report was named a “Best of ASH” presentation. Background Pearson Syndrome (PS)

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