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The Barth Syndrome Foundation Delivers Petition To FDA Advocating For A Fair, Equitable And Appropriate Review Of The Only Potential Treatment For Bar
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Arizona toddler with rare disorder could lose life-saving therapy
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Arizona toddler with rare disorder could lose life-saving therapy
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In a Nature Metabolism paper published today, researchers from the University of Pittsburgh detail a potential new target and a small-molecule drug candidate for treating Barth syndrome, a rare, life-threatening and currently incurable genetic disease with devastating consequences.