PYRUVATE KINASE DEFICIENCY
Clinical trials for PYRUVATE KINASE DEFICIENCY explained in plain language.
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Gene therapy may free patients from lifelong blood transfusions
Disease control CompletedThis early-stage trial tested a gene therapy for people with pyruvate kinase deficiency, a rare blood disorder that often requires frequent blood transfusions. Four adults and children received their own blood stem cells modified to carry a working copy of the faulty gene. The ma…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE1 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated May 26, 2026 11:09 UTC
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Promising pill may ease rare anemia without transfusions
Disease control CompletedThis study tested a drug called mitapivat in 52 adults with pyruvate kinase deficiency, a rare genetic disorder that causes red blood cells to break down too quickly, leading to anemia. The goal was to see if different doses of the drug could safely raise hemoglobin levels and re…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 19, 2026 11:47 UTC
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New hope for rare blood disorder: Long-Term drug study shows promise
Disease control CompletedThis study looked at the long-term safety and effectiveness of the drug mitapivat in 90 adults with pyruvate kinase deficiency, a rare genetic blood disorder that causes red blood cells to break down too quickly. Participants had already completed an earlier mitapivat study and c…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 18, 2026 12:03 UTC