PYRUVATE KINASE DEFICIENCY
Clinical trials for PYRUVATE KINASE DEFICIENCY explained in plain language.
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Extended hope: Long-Term drug study for rare blood disorder
Disease control CompletedThis study aimed to understand the long-term safety and effectiveness of the drug mitapivat for adults with pyruvate kinase deficiency, a rare genetic blood disorder. It was an extension study for 90 participants who had already benefited from mitapivat in earlier trials. The mai…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 16, 2026 15:26 UTC
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First patients receive One-Time gene therapy for rare blood disorder
Disease control CompletedThis early-stage study tested the safety of a one-time gene therapy for adults and children with pyruvate kinase deficiency, a rare genetic blood disorder. Doctors collected patients' own blood stem cells, corrected the faulty gene in a lab, and then infused the cells back. The m…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE1 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Mar 16, 2026 15:26 UTC