PYRUVATE KINASE DEFICIENCY
Clinical trials for PYRUVATE KINASE DEFICIENCY explained in plain language.
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Gene therapy offers hope for rare blood disorder patients
Disease control CompletedThis early-stage study tested a gene therapy for pyruvate kinase deficiency, a rare genetic blood disorder that causes severe anemia and often requires frequent blood transfusions. Four adults and children received their own blood stem cells modified to carry a working copy of th…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE1 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated May 17, 2026 11:50 UTC
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New hope for rare blood disorder: experimental drug shows promise in phase 2 trial
Disease control CompletedThis study tested a drug called mitapivat (AG-348) in 52 adults with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The goal was to see if different doses of the drug are safe and can help control the…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 17, 2026 11:36 UTC
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New hope for rare blood disorder: Long-Term drug study shows promise
Disease control CompletedThis study tested 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 earlier studies of mitapivat and co…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 11, 2026 20:53 UTC