Agios Pharmaceuticals, Inc. (AGIO) on Thursday announced positive results from its Phase 3 ACTIVATE-Kids study of mitapivat or PYRUKYND in children with pyruvate kinase deficiency who are not regularly transfused.
Pyruvate kinase (PK) deficiency is a rare condition caused by inherited mutations in the PKLR gene, leading to low energy and shorter lifespan of red blood cells. People with PK deficiency may have symptoms like anemia, extreme tiredness, trouble exercising, yellowing of the skin, memory problems, and difficulty focusing.
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