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Journal Articles

Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency

Eduard J. van Beers, Hanny Al-Samkari, Rachael F. Grace, Wilma Barcellini, Andreas Glenthøj, Melissa Dibacco, Penelope A. Kosinski, Emily Xu, Vanessa Beynon, Parija Patel, John B. Porter, Kevin H.M. Kuo

Blood (2022) 140 (Supplement 1): 2460–2462.

https://doi.org/10.1182/blood-2022-169098

Published: 2022

Journal Articles

Mitapivat Improves Markers of Erythropoietic Activity in Long-Term Study of Adults with Alpha- or Beta-Non-Transfusion-Dependent Thalassemia

Kevin H.M. Kuo, D. Mark Layton, Ashutosh Lal, Hanny Al-Samkari, Penelope A. Kosinski, Bo Tong, Jeremie H. Estepp, Katrin Uhlig, Elliott P. Vichinsky

Blood (2022) 140 (Supplement 1): 2479–2480.

https://doi.org/10.1182/blood-2022-163493

Published: 2022

Journal Articles

Activating Pyruvate Kinase Improves Red Blood Cell Integrity By Reducing Band3 Tyrosine Phosphorylation

Kang Le, Xunde Wang, Maureen Lundt, Anna Conrey, Ingrid Frey, Silvia Giannini, Penelope A. Kosinski, John A. Hausman, Philip S. Low, Neal Jeffries, Swee Lay Thein

Blood (2022) 140 (Supplement 1): 5395–5396.

https://doi.org/10.1182/blood-2022-160378

Published: 2022

Journal Articles

Pklr Variants Associated with Acute Pain in Sickle Cell Disease Influence ATP Concentrations in Red Blood Cells

Xunde Wang, Yi-Ping Fu, Quan Li, Emily B Dunkelberger, Mickias Tegegn, Penelope A. Kosinski, Heidi Mangus, John Hamre, Sam M. Mbulaiteye, Ludmila Prokunina-Olsson, Stephen J. Chanock, Nancy Asomaning, Neal Jeffries, William Eaton, Swee Lay Thein

Blood (2022) 140 (Supplement 1): 954–956.

https://doi.org/10.1182/blood-2022-167296

Published: 2022

Journal Articles

AG-946 Normalizes Glycolysis and Improves Red Cell Indices in a Humanized Sickle Cell Mouse Model

Rohitash Jamwal, Lily C. Wain, Christopher Copeland, Penelope A. Kosinski, Megan Wind-Rotolo, Ilya Gertsman, William Eaton, David M Bodine, Lenny Dang, Swee Lay Thein

Blood (2022) 140 (Supplement 1): 950–951.

https://doi.org/10.1182/blood-2022-156591

Published: 2022

Journal Articles

ACTIVATE-Kids: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused

Rachael F. Grace, Patrick D. Tyler, Erin Larcom, Penelope A. Kosinski, Vanessa Beynon

Blood (2022) 140 (Supplement 1): 5331–5332.

https://doi.org/10.1182/blood-2022-168652

Published: 2022

Journal Articles

ACTIVATE-KidsT: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Regularly Transfused

Rachael F. Grace, Patrick D. Tyler, Meredith Little, Penelope A. Kosinski, Vanessa Beynon

Blood (2022) 140 (Supplement 1): 5319–5320.

https://doi.org/10.1182/blood-2022-167452

Published: 2022

Journal Articles

Long-Term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat

Blood (2022) 140 (Supplement 1): 5313–5315.

https://doi.org/10.1182/blood-2022-169125

Published: 2022

Journal Articles

A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease

Blood (2022) 140 (19): 2053–2062.

https://doi.org/10.1182/blood.2022015403

Published: 2022

Includes: Supplemental data

Journal Articles

Pharmacodynamic Effects of AG-946, a Highly Potent Next-Generation Activator of Pyruvate Kinase, in Ex Vivo Treatment of Red Blood Cells from Sickle Cell Disease Patients

Minke A.E. Rab, Myrthe J. Van Dijk, Jennifer Bos, Brigitte A. van Oirschot, Johan Gerrits, Penelope A. Kosinski, Charles Kung, Judith J.M. Jans, Eduard J. Van Beers, Lenny Dang, Richard van Wijk

Blood (2021) 138 (Supplement 1): 2029.

https://doi.org/10.1182/blood-2021-145374

Published: 2021

Journal Articles

Mitapivat Improves Transfusion Burden and Reduces Iron Overload in Thalassemic Mice

Alessandro Mattè, Penelope A. Kosinski, Enrica Federti, Lenny Dang, Angela Siciliano, Iana Iatcenko, Carlo Brugnara, Lucia De Franceschi

Blood (2021) 138 (Supplement 1): 2016.

https://doi.org/10.1182/blood-2021-153721

Published: 2021

Journal Articles

Mitapivat Improves Ineffective Erythropoiesis and Reduces Iron Overload in Patients with Pyruvate Kinase Deficiency

Eduard J. Van Beers, Hanny Al-Samkari, Rachael F. Grace, Wilma Barcellini, Andreas Glenthoej, Malia P. Judge, Penelope A. Kosinski, Emily Xu, Vanessa Beynon, Bryan McGee, John B. Porter, Kevin H.M. Kuo

Blood (2021) 138 (Supplement 1): 2005.

https://doi.org/10.1182/blood-2021-147317

Published: 2021

Journal Articles

Mitapivat (AG-348) Demonstrates Safety, Tolerability, and Improvements in Anemia, Hemolysis, Oxygen Affinity, and Hemoglobin S Polymerization Kinetics in Adults with Sickle Cell Disease: A Phase 1 Dose Escalation Study

Julia Z. Xu, Anna Conrey, Ingrid Frey, Eveline Gwaabe, Laurel A Menapace, Laxminath Tumburu, Maureen Lundt, Quan Li, Kristen Glass, Varsha Iyer, Heidi Mangus, Charles Kung, Lenny Dang, Penelope A. Kosinski, Peter Hawkins, Neal Jeffries, William Eaton, Swee Lay Thein

Blood (2021) 138 (Supplement 1): 10.

https://doi.org/10.1182/blood-2021-145398

Published: 2021

Journal Articles

Long-Term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia

Kevin H.M. Kuo, D. Mark Layton, Ashutosh Lal, Hanny Al-Samkari, Joy Bhatia, Penelope A. Kosinski, Bo Tong, Megan Lynch, Katrin Uhlig, Elliott Vichinsky

Blood (2021) 138 (Supplement 1): 576.

https://doi.org/10.1182/blood-2021-150386

Published: 2021

Journal Articles

Durability of Hemoglobin Response and Reduction in Transfusion Burden Is Maintained over Time in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat in a Long-Term Extension Study

Blood (2021) 138 (Supplement 1): 848.

https://doi.org/10.1182/blood-2021-147711

Published: 2021

Journal Articles

Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy

Minke A. E. Rab, Jennifer Bos, Brigitte A. van Oirschot, Stephanie van Straaten, Penelope A. Kosinski, Victor Chubukov, Hyeryun Kim, Heidi Mangus, Roger E. G. Schutgens, Gerard Pasterkamp, Lenny Dang, Charles Kung, Eduard J. van Beers, Richard van Wijk

Blood (2021) 137 (21): 2997–3001.

https://doi.org/10.1182/blood.2020008635

Published: 2021

Includes: Supplemental data

Journal Articles

The Pyruvate Kinase Activator Mitapivat Ameliorates Anemia and Prevents Iron Overload in a Mouse Model of Hereditary Spherocytosis

Alessandro Mattè, BSc,PhD, Wilson Anand, Enrica Federti, PhD, Charles Kung, PhD, Penelope A. Kosinski, MS, Veronica Riccardi, Iana Iatcenko, Lenny Dang, PhD, Christophe Leboeuf, Anne Janin, PhD, Carlo Brugnara, MD, Mohandas Narla, DSc, Lucia De Franceschi, MD

Blood (2020) 136 (Supplement 1): 29.

https://doi.org/10.1182/blood-2020-142232

Published: 2020

Journal Articles

Phase 1 Multiple Ascending Dose Study of Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Mitapivat (AG-348) in Subjects with Sickle Cell Disease

Blood (2020) 136 (Supplement 1): 21–22.

https://doi.org/10.1182/blood-2020-137716

Published: 2020

Journal Articles

Decreased Activity and Stability of Pyruvate Kinase in Hereditary Hemolytic Anemia: A Potential Target for Therapy By AG-348 (Mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase

Minke A.E. Rab, MD, Brigitte A. van Oirschot, Stephanie van Straaten, MD PhD, Bart J. Biemond, MD PhD, Jennifer Bos, Penelope A. Kosinski, MS, Charles Kung, PhD, Eduard J. van Beers, MD PhD, Richard van Wijk, PhD

Blood (2019) 134 (Supplement_1): 3506.

https://doi.org/10.1182/blood-2019-129996

Published: 2019

Journal Articles

Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency

Kathryn Addonizio, BA, Hanny Al-Samkari, MD, Bertil Glader, MD PhD, D Holmes Morton, MD, Satheesh Chonat, MD, Alexis A. Thompson, MD MPH, Kevin H. M. Kuo, MDMSc,FRCPC, Yaddanapudi Ravindranath, MBBS, Heng Wang, MD PhD, Jennifer A. Rothman, MD, Janet L. Kwiatkowski, MD MSCE, Charles Kung, PhD, Penelope A. Kosinski, MS, Hasan Al-Sayegh, MS, Wendy B. London, PhD, Rachael F. Grace, MD

Blood (2019) 134 (Supplement_1): 3515.

https://doi.org/10.1182/blood-2019-125248

Published: 2019

NARROW

Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency

Mitapivat Improves Markers of Erythropoietic Activity in Long-Term Study of Adults with Alpha- or Beta-Non-Transfusion-Dependent Thalassemia

Activating Pyruvate Kinase Improves Red Blood Cell Integrity By Reducing Band3 Tyrosine Phosphorylation

Pklr Variants Associated with Acute Pain in Sickle Cell Disease Influence ATP Concentrations in Red Blood Cells

AG-946 Normalizes Glycolysis and Improves Red Cell Indices in a Humanized Sickle Cell Mouse Model

ACTIVATE-Kids: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused

ACTIVATE-KidsT: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Regularly Transfused

Long-Term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat

A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease

Pharmacodynamic Effects of AG-946, a Highly Potent Next-Generation Activator of Pyruvate Kinase, in Ex Vivo Treatment of Red Blood Cells from Sickle Cell Disease Patients

Mitapivat Improves Transfusion Burden and Reduces Iron Overload in Thalassemic Mice

Mitapivat Improves Ineffective Erythropoiesis and Reduces Iron Overload in Patients with Pyruvate Kinase Deficiency

Mitapivat (AG-348) Demonstrates Safety, Tolerability, and Improvements in Anemia, Hemolysis, Oxygen Affinity, and Hemoglobin S Polymerization Kinetics in Adults with Sickle Cell Disease: A Phase 1 Dose Escalation Study

Long-Term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia

Durability of Hemoglobin Response and Reduction in Transfusion Burden Is Maintained over Time in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat in a Long-Term Extension Study

Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy

The Pyruvate Kinase Activator Mitapivat Ameliorates Anemia and Prevents Iron Overload in a Mouse Model of Hereditary Spherocytosis

Phase 1 Multiple Ascending Dose Study of Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Mitapivat (AG-348) in Subjects with Sickle Cell Disease

Decreased Activity and Stability of Pyruvate Kinase in Hereditary Hemolytic Anemia: A Potential Target for Therapy By AG-348 (Mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase

Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency

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American Society of Hematology

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American Society of Hematology

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