PEDIATRIC HEMOLYTIC ANEMIA
Clinical trials for PEDIATRIC HEMOLYTIC ANEMIA explained in plain language.
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Hope for kids with rare anemia: drug may cut need for transfusions
Disease control OngoingThis study tests an experimental drug called 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. The goal is to see if mitapivat can safely reduce the number of blood transfusions these…
Matched conditions: PEDIATRIC HEMOLYTIC ANEMIA
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 17, 2026 10:50 UTC
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New hope for kids with rare blood disorder: pill may reduce need for transfusions
Disease control OngoingThis study tests an oral drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic anemia that causes red blood cells to break down too quickly. The goal is to see if the drug can raise hemoglobin levels and reduce the need for blood transfusi…
Matched conditions: PEDIATRIC HEMOLYTIC ANEMIA
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 15, 2026 11:53 UTC