PEDIATRIC HEMOLYTIC ANEMIA
Clinical trials for PEDIATRIC HEMOLYTIC ANEMIA explained in plain language.
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New pill offers hope for kids with rare, debilitating blood disease
Disease control OngoingThis study is testing whether an oral drug called mitapivat is safe and effective for children with pyruvate kinase deficiency (PKD), a rare genetic disorder that causes chronic anemia. About 30 children, aged 1 to 18, who do not regularly need blood transfusions will take either…
Matched conditions: PEDIATRIC HEMOLYTIC ANEMIA
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC
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New hope for kids tied to blood transfusions: trial aims to cut lifeline need
Disease control OngoingThis study is testing whether a drug called mitapivat can help children with pyruvate kinase deficiency (PK deficiency), a rare genetic disorder that destroys red blood cells, rely less on regular blood transfusions. About 49 children, aged 1 to 18, who currently need frequent tr…
Matched conditions: PEDIATRIC HEMOLYTIC ANEMIA
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 18, 2026 14:40 UTC