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Agios Pharma (AGIO) to complete Phase 1 study of AG-519 in Patients With Pyruvate Kinase Deficiency in September 2016

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Additional Relevant Details PK deficiency disease understanding is still evolving. The disease typically presents during early infancy with jaundice and severe anemia, which can require immediate life-saving intervention via replacement of the infant’s entire blood system with a donor’s blood, referred to as an exchange transfusion. The current standard of care for PK deficiency is supportive, including blood transfusions, splenectomy, chelation therapy to address iron overload and/or interventions for other treatment- and disease-related morbidities. Currently, there is no approved therapy to treat the underlying cause of PK deficiency.
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Jun 09, 2016
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Related Keywords Pyruvate Kinase Deficiency, Pk Deficiency, Phase 1 Study, Ag-519, Double Blind