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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 metabolic enzyme pyruvate kinase M2 regulates platelet function and arterial thrombosis

Manasa K. Nayak, Madankumar Ghatge, Gagan D. Flora, Nirav Dhanesha, Manish Jain, Kathleen R. Markan, Matthew J. Potthoff, Steven R. Lentz, Anil K. Chauhan

Blood (2021) 137 (12): 1658–1668.

https://doi.org/10.1182/blood.2020007140

Published: 2021

Includes: Supplemental data

Journal Articles

Phenotypic Corrections of Ex Vivo Erythrocytes Derived from Chronic Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 ( KLF-1 ) Mutations By AG-348: A Novel Synthetic Pyruvate Kinase Activator

Thidarat Suksangpleng, PhD, Kalaya Tachavanich, M.Sc., Suchada Riolueang, MSc, Supachai Ekwattanakit, PhDMD, Vip Viprakasit, MD DPhil

Blood (2019) 134 (Supplement_1): 2220.

https://doi.org/10.1182/blood-2019-131720

Published: 2019

Journal Articles

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Kevin H. M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB BS, Katrin Uhlig, MD MS, Megan Lynch, MSN, NP, Li Liu, PhD, Elliott P. Vichinsky, MD

Blood (2019) 134 (Supplement_1): 2249.

https://doi.org/10.1182/blood-2019-123113

Published: 2019

Journal Articles

Hereditary Hemolytic Anemias Other Than Thalassemia: Single Center Experience

Gul Nihal Ozdemir, MD, Mehmet Akif Kilic, MD

Blood (2019) 134 (Supplement_1): 4855.

https://doi.org/10.1182/blood-2019-121702

Published: 2019

Journal Articles

Pyruvate Kinase Deficiency Cases Mimicking Congenital Dyserythropoietic Anemia

Sule Unal, MD, Tekin Aksu, MD, Cagrı Coskun, MD, Fatma Gumruk, MD

Blood (2019) 134 (Supplement_1): 4794.

https://doi.org/10.1182/blood-2019-124272

Published: 2019

Journal Articles

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study (ACTIVATE) in Progress

Eduard J. van Beers, MD PhD, Malia P. Judge, BS, RN, Lei Hua, PhD, Chris Mix, MD

Blood (2019) 134 (Supplement_1): 4791.

https://doi.org/10.1182/blood-2019-123100

Published: 2019

Journal Articles

Phase 1 Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, in Healthy and Sickle Cell Disease Subjects

Theodosia A. Kalfa, MD PhD, Frans A Kuypers, PhD, Marilyn J. Telen, MD, Punam Malik, MD, Diamantis G. Konstantinidis, PhD BSc, Jeremy H. Estepp, MD, Hyon J. Kim, MD PhD, Santosh L. Saraf, MD, Lindsey Wilson, Maria D Ribadeneira, PhD, Sanjeev Forsyth, Patricia Schroeder, Adam Drake, Olga Polyanskaya, Patrick Kelly, MD, Lukasz Biernat, MD

Blood (2019) 134 (Supplement_1): 616.

https://doi.org/10.1182/blood-2019-121889

Published: 2019

Journal Articles

Targeting Metabolic Enzyme Pyruvate Kinase M2: A Novel Strategy to Inhibit Platelet Function and Arterial Thrombosis

Manasa K Nayak, PhD, Madankumar Ghatge, PhD, Nirav Dhanesha, PhD, Gagan D Flora, PhD, Manish Jain, PhD, Omar Rodriguez, MD BS, Kathleen Markan, PhD, Mathhhew Potthoff, PhD, Steven R. Lentz, MD PhD, Anil K Chauhan, PhD

Blood (2019) 134 (Supplement_1): 1056.

https://doi.org/10.1182/blood-2019-129027

Published: 2019

Journal Articles

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

Blood (2019) 134 (Supplement_1): 2175.

https://doi.org/10.1182/blood-2019-123069

Published: 2019

Journal Articles

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused: A Phase 3, Open-Label, Multicenter, Study (ACTIVATE-T) in Progress

Megan Lynch, MSN, Lei Hua, PhD, Chris Mix, MD, John B. Porter, MA MDFRCP FRCPath

Blood (2019) 134 (Supplement_1): 3526.

https://doi.org/10.1182/blood-2019-123107

Published: 2019

Journal Articles

Development of Patient-Reported Outcome Measures (Symptoms and Impacts) in Adults with Pyruvate Kinase Deficiency

Sam Salek, PhDRPh FFPM MCMS MRPSGB FESCP, Audra N. Boscoe, PhD, Chris Evans, PhD, Shayna Egan, MPH, Ted Wells, Sarah Piantedosi, Jennifer Cohen, Robert J Klaassen, MD FRCPC, Rachael F. Grace, MD, Mike Storm, MSc

Blood (2019) 134 (Supplement_1): 3447.

https://doi.org/10.1182/blood-2019-128524

Published: 2019

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

Journal Articles

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

Rachael F. Grace, MD, D. Mark Layton, MB BS, FRCP, FRCPCH, Frédéric Galactéros, MD PhD, Wilma Barcellini, MD, Eduard J. van Beers, MD PhD, Hassan M. Yaish, MD, Yaddanapudi Ravindranath, MBBS, Kevin H. M. Kuo, MDMSc,FRCPC, Sujit Sheth, MD, Janet L. Kwiatkowski, MD MSCE, Lei Hua, PhD, Peter F. Hawkins, PhD, Chris Mix, MD, Bertil Glader, MD PhD

Blood (2019) 134 (Supplement_1): 3512.

https://doi.org/10.1182/blood-2019-123406

Published: 2019

Journal Articles

The Effect of Pyruvate Kinase M2: Inhibitor(shikonin) on the Function of Mdc in Severe Aplastic Anemia Mouse Model

Mengying Zheng, Chunyan Liu, Yuanyuan Shao, Rong Fu, Jr., PhD, Huaquan Wang, MD, Ting Wang, Weiwei Qi, Zonghong Shao

Blood (2019) 134 (Supplement_1): 5021.

https://doi.org/10.1182/blood-2019-126726

Published: 2019

Journal Articles

An Ongoing Global, Longitudinal, Observational Study of Patients with Pyruvate Kinase Deficiency: The PEAK Registry

Rachael F. Grace, MD, Paola Bianchi, BSc, PhD, Bertil Glader, MD PhD, Andreas Glenthøj, MD PhD, Bryan Jones, PhD, Hitoshi Kanno, MD PhD, Kevin H. M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB ChB, Eduard J. van Beers, MD PhD, Joan-Lluis Vives Corrons, MD PhD, Tu Xu, MS, PhD

Blood (2019) 134 (Supplement_1): 2223.

https://doi.org/10.1182/blood-2019-123059

Published: 2019

Journal Articles

Epigenetic Activation of the pH Regulator MCT4 in Acute Myeloid Leukemia Exploits a Fundamental Metabolic Process of Enhancing Cell Growth through Proton Shifting

Cheuk-Him Man, David T. Scadden, MD, Francois Mercier, MD FRCPC, Nian Liu, Wentao Dong, Gregory Stephanopoulos, PhD, Li Jiang, PhD, Yookyung Jung, PhD, Charles Lin, PhD, Anskar Y.-H. Leung, MD PhD

Blood (2019) 134 (Supplement_1): 3765.

https://doi.org/10.1182/blood-2019-123604

Published: 2019

Journal Articles

Tibetan Enriched PKLR Variant Is Beneficial to High Altitude Adaption By Improving Oxygen Delivery

Jihyun Song, PhD, Virginia Abello, MD, Ricardo Amaru, MD, Adelina Sergueeva, MD, Jainagul Isakova, Penelope A. Kosinski, MS, Brigitte A. van Oirschot, Victor R. Gordeuk, MD, Charles Kung, PhD, Richard van Wijk, PhD, Josef T. Prchal, MD

Blood (2018) 132 (Supplement 1): 1027.

https://doi.org/10.1182/blood-2018-99-117389

Published: 2018

Journal Articles

Efficient CRISPR/Cas9-Mediated Gene Editing of Pklr in Human Hematopoietic Progenitors and Stem Cells for the Gene Therapy of Pyruvate Kinase Deficiency

Oscar Quintana Bustamante, PhD, Sara Fañanas-Baquero, Daniel P. Dever, PhD, Alberquilla Omaira, Joab Camarena, BS, Rebeca Sanchez-Dominguez, PhD, Matias Morin, Val Fernandez, Miguel Angel Moreno-Pelayo, Juan A. Bueren, PhD, Matthew Porteus, MD PhD, Jose C Segovia, PhD

Blood (2018) 132 (Supplement 1): 5792.

https://doi.org/10.1182/blood-2018-99-111772

Published: 2018

NARROW

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

The metabolic enzyme pyruvate kinase M2 regulates platelet function and arterial thrombosis

Phenotypic Corrections of Ex Vivo Erythrocytes Derived from Chronic Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 ( KLF-1 ) Mutations By AG-348: A Novel Synthetic Pyruvate Kinase Activator

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Hereditary Hemolytic Anemias Other Than Thalassemia: Single Center Experience

Pyruvate Kinase Deficiency Cases Mimicking Congenital Dyserythropoietic Anemia

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study (ACTIVATE) in Progress

Phase 1 Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, in Healthy and Sickle Cell Disease Subjects

Targeting Metabolic Enzyme Pyruvate Kinase M2: A Novel Strategy to Inhibit Platelet Function and Arterial Thrombosis

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused: A Phase 3, Open-Label, Multicenter, Study (ACTIVATE-T) in Progress

Development of Patient-Reported Outcome Measures (Symptoms and Impacts) in Adults with Pyruvate Kinase Deficiency

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

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

The Effect of Pyruvate Kinase M2: Inhibitor(shikonin) on the Function of Mdc in Severe Aplastic Anemia Mouse Model

An Ongoing Global, Longitudinal, Observational Study of Patients with Pyruvate Kinase Deficiency: The PEAK Registry

Epigenetic Activation of the pH Regulator MCT4 in Acute Myeloid Leukemia Exploits a Fundamental Metabolic Process of Enhancing Cell Growth through Proton Shifting

Tibetan Enriched PKLR Variant Is Beneficial to High Altitude Adaption By Improving Oxygen Delivery

Efficient CRISPR/Cas9-Mediated Gene Editing of Pklr in Human Hematopoietic Progenitors and Stem Cells for the Gene Therapy of Pyruvate Kinase Deficiency

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American Society of Hematology

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American Society of Hematology

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