Records containing: Pyruvate Kinase Deficiency
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CLINICAL_TRIALS study record update · 2026-04-23Pyruvate Kinase Deficiency Global Longitudinal Registry
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CLINICAL_TRIALS study record update · 2026-04-16A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Subjects With Pyruvate Kinase Deficiency
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CLINICAL_TRIALS study record update · 2026-03-30A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
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CLINICAL_TRIALS study record update · 2026-03-25Characterization Of Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
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CLINICAL_TRIALS study record update · 2025-11-18An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies
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CLINICAL_TRIALS study record update · 2025-09-05Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects With PKD
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CLINICAL_TRIALS study record update · 2023-10-16Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008: Cognition in Participants With Pyruvate Kinase Deficiency
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CLINICAL_TRIALS study record update · 2023-09-07Pyruvate Kinase Deficiency Global Longitudinal Registry: Patient-Reported Outcomes
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CLINICAL_TRIALS study record update · 2023-02-09Pyruvate Kinase Deficiency Epidemiological Study. An International, Multicentre, Epidemiological Observational Study
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CLINICAL_TRIALS study record update · 2022-05-24A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Subjects With Pyruvate Kinase Deficiency
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CLINICAL_TRIALS study record update · 2021-12-10An Open-Label Study To Evaluate the Efficacy and Safety of AG-348 in Regularly Transfused Adult Subjects With Pyruvate Kinase (PK) Deficiency
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CLINICAL_TRIALS study record update · 2020-06-11A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency
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CLINICAL_TRIALS study record update · 2020-05-22Pyruvate Kinase Deficiency (PKD) Natural History Study