ALLMedicine™ Pyruvate Kinase Deficiency Center

Mar 10th, 2023 - Pyruvate kinase deficiency (PKD) is an autosomal recessive condition, caused due to homozygous or compound heterozygous mutation in the PKLR gene resulting in non-spherocytic hereditary hemolytic anemia. Clinical manifestations in PKD patients var...

Feb 28th, 2023 - This is a multicenter, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with AG-348 in participants who were previously enrolled in Study AG348-C-006 or Study AG348-C-007.

Feb 28th, 2023 - Data will be submitted to the Registry via electronic case report forms (eCRFs). Relevant datasets, such as historical trial data, claims, medical records, or central lab data will be electronically integrated into the Registry or Registry reporti...

Feb 17th, 2023 - ACTIVATE-KidsT (AG348-C-022) is a multicenter study designed to evaluate the efficacy and safety of treatment with mitapivat compared with placebo in pediatric participants with pyruvate kinase deficiency (PK deficiency) who are regularly receivin...

Feb 17th, 2023 - Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving blood transfusions. P...

Oct 26th, 2018 - Pyruvate kinase deficiency (PKD) is the most common enzyme defect of glycolysis and an important cause of hereditary, nonspherocytic hemolytic anemia. The disease has a worldwide geographical distribution but there are no verified data regarding i...

Feb 28th, 2023 - This is a multicenter, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with AG-348 in participants who were previously enrolled in Study AG348-C-006 or Study AG348-C-007.

Feb 28th, 2023 - Data will be submitted to the Registry via electronic case report forms (eCRFs). Relevant datasets, such as historical trial data, claims, medical records, or central lab data will be electronically integrated into the Registry or Registry reporti...

Feb 17th, 2023 - Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving blood transfusions. P...

Feb 17th, 2023 - ACTIVATE-KidsT (AG348-C-022) is a multicenter study designed to evaluate the efficacy and safety of treatment with mitapivat compared with placebo in pediatric participants with pyruvate kinase deficiency (PK deficiency) who are regularly receivin...

Feb 10th, 2023 - This is a substudy of Study AG348-C-008, an observational, longitudinal, multicenter, global registry that aims to better understand the longitudinal clinical implications of pyruvate kinase deficiency (PK deficiency). The primary purpose of this ...

Apr 14th, 2022 - Treatment with mitapivat (Pyrukynd) increased hemoglobin levels and decreased hemolysis in patients with pyruvate kinase (PK) deficiency who did not require regular blood transfusions, the randomized ACTIVATE trial showed. For the phase III study'...

Feb 18th, 2022 - Note that some links may require registration or subscription. Researchers argued that BA.2 should not be considered a subvariant of Omicron based on lab data hinting at greater severity: "It looks like we might be looking at a new Greek letter he...

Oct 30th, 2020 - Pyruvate kinase deficiency is an inherited metabolic disorder of the enzyme pyruvate kinase, which affects the survival of erythrocytes. Both autosomal dominant and recessive inheritance have been observed with the disorder; classically, and more ...

Sep 4th, 2019 - Mitapivat showed positive safety and efficacy outcomes in patients with pyruvate kinase deficiency, according to results from a phase 2 trial. After 24 weeks of treatment, the therapy was associated with a rapid rise in hemoglobin levels in 50% of.

Sep 4th, 2019 - Mitapivat showed positive safety and efficacy outcomes in patients with pyruvate kinase deficiency, according to results from a phase 2 trial. After 24 weeks of treatment, the therapy was associated with a rapid rise in hemoglobin levels in 50% of.