PYRUVATE KINASE DEFICIENCY
Clinical trials for PYRUVATE KINASE DEFICIENCY explained in plain language.
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Lifeline drug mitapivat keeps flowing for rare blood disorder patients
Disease control ENROLLING_BY_INVITATIONThis study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does …
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE4 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
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Gene therapy offers hope for rare blood disorder
Disease control TerminatedThis study tests a gene therapy for people with pyruvate kinase deficiency, a rare blood disorder causing severe anemia. Ten participants will receive their own blood stem cells modified with a healthy gene to help produce normal red blood cells. The goal is to raise hemoglobin l…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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Global registry launches to unlock secrets of rare blood disorder
Knowledge-focused OngoingThis study is a global registry that will follow about 500 people with pyruvate kinase (PK) deficiency, a rare inherited anemia, for up to 9 years. Researchers will collect medical data to better understand the disease's natural history, treatments, and complications. No new drug…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Sponsor: Agios Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:02 UTC