PEDIATRIC PYRUVATE KINASE DEFICIENCY
Clinical trials for PEDIATRIC PYRUVATE KINASE DEFICIENCY explained in plain language.
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Could a pill replace blood transfusions for kids with rare anemia?
Disease control OngoingThis phase 3 trial tests the drug mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic disorder that causes red blood cells to break down too quickly. These children need regular blood transfusions. The study compares mitapivat to a placebo to see if…
Matched conditions: PEDIATRIC PYRUVATE KINASE DEFICIENCY
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
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Could a daily pill help kids with rare blood disorder?
Disease control OngoingThis study tests a drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The trial compares mitapivat to a placebo to see if it can raise hemoglobin levels…
Matched conditions: PEDIATRIC PYRUVATE KINASE DEFICIENCY
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC