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Journal Articles

Congenital anemia reveals distinct targeting mechanisms for master transcription factor GATA1

Blood (2022) 139 (16): 2534–2546.

DOI: https://doi.org/10.1182/blood.2021013753

Published: 2022

Includes: Supplemental data

Journal Articles

Targeting of Calbindin 1 (CALB1) Rescues Erythropoiesis in a Human Model of Diamond Blackfan Anemia: Implications for Novel Therapies

Nan of Wang, PhD, Corinne Marie Lavasseur, BA, Hongxia Yan, MS, Scott Younger, PhD, Bertil Glader, MD PhD, Kathleen M. Sakamoto, MD PhD, Lionel Blanc, PhD, Anupama Narla, MD

Blood (2020) 136 (Supplement 1): 4.

DOI: https://doi.org/10.1182/blood-2020-139823

Published: 2020

Journal Articles

SATB1 Regulates GATA1 Protein Expression in Early Hematopoiesis and Is Deregulated in Diamond Blackfan Anemia

Mark C Wilkes, PhD, Hee-Don Chae, PhD, Ethan Patrick Wentworth, Toshinobu Nishimura, PhD, Anupama Narla, MD, Bertil Glader, MD PhD, Hiromitsu Nakauchi, Joanna Wysocka, Kathleen M. Sakamoto, MD PhD

Blood (2020) 136 (Supplement 1): 3.

DOI: https://doi.org/10.1182/blood-2020-140437

Published: 2020

Journal Articles

Baseline Characteristics of Patients in Peak: A Global, Longitudinal Registry of Patients with Pyruvate Kinase Deficiency

Rachael F. Grace, MD, Audra Boscoe, PhD, Chris Bowden, MD, Bertil Glader, MD PhD, Hitoshi Kanno, MDPhD, D. Mark Layton, MB BS, Eduard van Beers, MD PhD, Joan-Lluis Vives Corrons, MD PhD, Yan Yan, MS, Paola Bianchi, BSc, PhD

Blood (2020) 136 (Supplement 1): 39–40.

DOI: https://doi.org/10.1182/blood-2020-136634

Published: 2020

Journal Articles

Metformin Upregulates Mir-26a to Improve Erythropoiesis in Preclinical Models of Diamond Blackfan Anemia through Suppression of Nlk Expression

Mark C Wilkes, PhD, Kavitha Siva, PhD, Jacqueline D Mercado, Ethan Patrick Wentworth, Mallika Saxena, Jun Chen, MD PhD, Anupama Narla, MD, Bertil Glader, MD PhD, Shuo Lin, PhD, Manuel Serrano, PhD, Johan Flygare, MD PhD, Kathleen M. Sakamoto, MD PhD

Blood (2020) 136 (Supplement 1): 7.

DOI: https://doi.org/10.1182/blood-2020-137170

Published: 2020

Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

Blood (2020) 136 (Supplement 1): 47.

DOI: https://doi.org/10.1182/blood-2020-137246

Published: 2020

Journal Articles

Pharmacological Inhibition of Nemo-like Kinase Rescues mTOR-Mediated Translation and Primes Progenitors for Leucine-Stimulated Erythroid Expansion in Pre-Clinical Models of Diamond Blackfan Anemia

Mark C Wilkes, PhD, Jacqueline D Mercado, Mallika Saxena, Jun Chen, MD PhD, Kavitha Siva, PhD, Hee-Don Chae, PhD, Minyoung Youn, PhD, Ethan Patrick Wentworth, Kevin Jung, Ryan Sathianathen, Hiral Patel, Bertil Glader, MD PhD, Anupama Narla, MD, Claire E Repellin, PhD, Hanna T Gazda, MD PhD, Manuel Serrano, PhD, Johan Flygare, MD PhD, Kathleen M. Sakamoto, MD PhD

Blood (2019) 134 (Supplement_1): 455.

DOI: https://doi.org/10.1182/blood-2019-129570

Published: 2019

Journal Articles

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

Blood (2019) 134 (Supplement_1): 949.

DOI: https://doi.org/10.1182/blood-2019-125352

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.

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

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.

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

Published: 2019

Journal Articles

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

Blood (2019) 134 (Supplement_1): 2175.

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

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.

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

Published: 2019

Journal Articles

Pharmacological Inhibition of Nlk (Nemo-like Kinase) Rescues Erythropoietic Defects in Pre-Clinical Models of Diamond Blackfan Anemia

Mark C Wilkes, Jun Chen, MD PhD, Kavitha Siva, PhD, Gianluca Veretti, PhD, Daniel P. Dever, PhD, Minyoung Youn, PhD, Hee-Don Chae, PhD, Jacqueline D Mercado, Mallika Saxena, Anupama Narla, MD, Bertil Glader, MD, Matthew Porteus, MD PhD, Claire E Repellin, PhD, Hanna T Gazda, MD PhD, Manuel Serrano, PhD, Johan Flygare, MD PhD, Kathleen M. Sakamoto, MD PhD

Blood (2018) 132 (Supplement 1): 754.

DOI: https://doi.org/10.1182/blood-2018-99-119954

Published: 2018

Journal Articles

Leucine for the Treatment of Transfusion Dependence in Patients with Diamond Blackfan Anemia

Adrianna Vlachos, MD, Evangelia Atsidaftos, MA, MPH, Ellen Muir, RN, MSN, Zora R. Rogers, MD, Mohammad Lufti Lababidi, MD, Waseem Alhushki, MD, Jason E. Farrar, MD, Bertil Glader, MD, Barbara Gruner, MD, Helge Hartung, MD, Christine M. Knoll, MD, Grzegorz Nalepa, MDPhD, Anupama Narla, MD, Arun Ranjan Panigrahi, MD, Colin A. Sieff, MB, BCh, Kelly J. Walkovich, MD, Jeffrey M Lipton, MD PhD

Blood (2018) 132 (Supplement 1): 755.

DOI: https://doi.org/10.1182/blood-2018-99-113570

Published: 2018

Journal Articles

Health Related Quality of Life and Fatigue in Patients with Pyruvate Kinase Deficiency

Blood (2018) 132 (Supplement 1): 4807.

DOI: https://doi.org/10.1182/blood-2018-99-113206

Published: 2018

Journal Articles

Pklr Intron Splicing-Associated Mutations and Alternate Diagnoses Are Common in Pyruvate Kinase Deficient Patients with Single or No Pklr Coding Mutations

Kimberly Lezon-Geyda, PhD, Melissa J. Rose, DO, Melissa A. McNaull, MD, Christine M. Knoll, MD, Hassan M. Yaish, MD, Yves D. Pastore, MD, Elisa Fermi, Bertil Glader, MD, Paola Bianchi, BSc, PhD, Rachael F. Grace, MD, Patrick G. Gallagher, MD

Blood (2018) 132 (Supplement 1): 3607.

DOI: https://doi.org/10.1182/blood-2018-99-117805

Published: 2018

Journal Articles

Genotype-Response Correlation in DRIVE PK, a Phase 2 Study of AG-348 in Patients with Pyruvate Kinase Deficiency

Charles Kung, PhD, Penelope A. Kosinski, MS, Heidi Mangus, BS, Lei Hua, PhD, Gary Connor, Michelle Mobilia, BS, Karen Sullivan, BA, Sasha Frye, BS, Marie-Hélène Jouvin, MD, Rachael F. Grace, MD, Bertil Glader, MD, Chris Bowden, MD

Blood (2018) 132 (Supplement 1): 3621.

DOI: https://doi.org/10.1182/blood-2018-99-111166

Published: 2018

Journal Articles

Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study

Blood (2018) 131 (20): 2183–2192.

DOI: https://doi.org/10.1182/blood-2017-10-810796

Published: 2018

Includes: Supplemental data

Journal Articles

Fetal Erythropoiesis Is Defective in Rpl11 Heterozygous Mice and Increases in Severity in Young Animals

Dolly Thomas, PhD, Cailin Joyce, PhD, Robert A. Redd, MS, Bertil Glader, MD, Steven Ellis, PhD, Donna S. Neuberg, ScD, Meaghan McGuinness, BS, Colin A. Sieff, MB, BCh, Carl D. Novina, MD PhD

Blood (2017) 130 (Suppl_1): 872.

DOI: https://doi.org/10.1182/blood.V130.Suppl_1.872.872

Published: 2017

Journal Articles

Nemo-like Kinase Hyper-Activated in Erythroid Progenitors in Models of Diamond Blackfan Anemia

Mark C Wilkes, Jae Wook Lee, Sriya Kudaravalli, Anupama Narla, MD, Bertil Glader, MD, Kathleen M. Sakamoto, MD PhD

Blood (2017) 130 (Suppl_1): 78.

DOI: https://doi.org/10.1182/blood.V130.Suppl_1.78.78

Published: 2017

NARROW

Congenital anemia reveals distinct targeting mechanisms for master transcription factor GATA1

Targeting of Calbindin 1 (CALB1) Rescues Erythropoiesis in a Human Model of Diamond Blackfan Anemia: Implications for Novel Therapies

SATB1 Regulates GATA1 Protein Expression in Early Hematopoiesis and Is Deregulated in Diamond Blackfan Anemia

Baseline Characteristics of Patients in Peak: A Global, Longitudinal Registry of Patients with Pyruvate Kinase Deficiency

Metformin Upregulates Mir-26a to Improve Erythropoiesis in Preclinical Models of Diamond Blackfan Anemia through Suppression of Nlk Expression

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

Pharmacological Inhibition of Nemo-like Kinase Rescues mTOR-Mediated Translation and Primes Progenitors for Leucine-Stimulated Erythroid Expansion in Pre-Clinical Models of Diamond Blackfan Anemia

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

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

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

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

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

Pharmacological Inhibition of Nlk (Nemo-like Kinase) Rescues Erythropoietic Defects in Pre-Clinical Models of Diamond Blackfan Anemia

Leucine for the Treatment of Transfusion Dependence in Patients with Diamond Blackfan Anemia

Health Related Quality of Life and Fatigue in Patients with Pyruvate Kinase Deficiency

Pklr Intron Splicing-Associated Mutations and Alternate Diagnoses Are Common in Pyruvate Kinase Deficient Patients with Single or No Pklr Coding Mutations

Genotype-Response Correlation in DRIVE PK, a Phase 2 Study of AG-348 in Patients with Pyruvate Kinase Deficiency

Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study

Fetal Erythropoiesis Is Defective in Rpl11 Heterozygous Mice and Increases in Severity in Young Animals

Nemo-like Kinase Hyper-Activated in Erythroid Progenitors in Models of Diamond Blackfan Anemia

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American Society of Hematology

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ASH Publications

American Society of Hematology

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