Pyruvate Kinase Deficiency
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period [Phase 3]
Phase 3 clinical trial to evaluate the efficacy and safety of mitapivat in pediatric patients with pyruvate kinase deficiency, including a 5…
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: Pyruvate Kinase Deficiency Global Longitudinal Registry
A global longitudinal registry for Pyruvate Kinase Deficiency sponsored by Agios Pharmaceuticals, with the study currently active but not re…
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CLINICAL_TRIALS research · 2026-03-30Clinical Trial Update: A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study [Phase 4]
This document outlines a Phase 4 study providing continued access to mitapivat for participants who previously completed an Agios-sponsored …
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CLINICAL_TRIALS research · 2026-03-25Clinical Trial Update: Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
This clinical trial update discusses a study of acquired pyruvate kinase deficiency in clonal myeloid neoplasms. The trial is currently acti…