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Journal Articles

Bone Marrow (BM) Delivery of Genetically-Modified (gm) Adult CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Improves Homing and Engraftment of Short-Term Progenitors over Long-Term Repopulating Hematopoietic Stem Cells

Sydney Felker, Archana Shrestha, Punam Malik, MD

Blood (2020) 136 (Supplement 1): 22–23.

https://doi.org/10.1182/blood-2020-138529

Published: 2020

Journal Articles

Rapid and Automated Quantitation of Dense Red Blood Cells: A Robust Biomarker of Therapeutic Response to Early Initiation of Hydroxyurea in Young Children with Sickle Cell Anemia

Alina Sadaf, MD, Charles T. Quinn, MD, Jennifer B Korpik, Amanda Pfeiffer, BS, MA, Mary Reynaud, MA, MT(ASCP), Omar Niss, MD, Punam Malik, MD, Russell E. Ware, MD PhD, Theodosia A. Kalfa, MD PhD, Patrick T. McGann, MD MS

Blood (2020) 136 (Supplement 1): 16–17.

https://doi.org/10.1182/blood-2020-137070

Published: 2020

Journal Articles

Oral Administration of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Has Potent Anti-Sickling Effects in a Sickle Cell Anemia (SCA) Mouse Model, Resulting in Improved RBC Survival and Hemoglobin Levels

Archana Shrestha, Mengna Chi, PhD, Kimberly Wagner, Adam Drake, Keertik Fulzele, PhD, Sylvie Guichard, Punam Malik, MD

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

https://doi.org/10.1182/blood-2020-140875

Published: 2020

Journal Articles

Angiotensin Signaling Is Essential for Stress Erythropoiesis but Results in Retention of Dysfunctional Mitochondria in Erythrocytes That Generate Excessive Reactive Oxygen Species

Parul Rai, MDMBBS, Swarnava Roy, PhD, Diamantis G. Konstantinidis, PhD BSc, Sithara Raju Ponny, MS, Marthe-sandrine Eiymo Mwa Mpollo, PhD, Archana Shrestha, Theodosia A. Kalfa, MD PhD, Punam Malik, MD

Blood (2020) 136 (Supplement 1): 31–32.

https://doi.org/10.1182/blood-2020-139980

Published: 2020

Journal Articles

Increased Hydroxyurea Prescribing Practices over Ten Years with Improved Clinical Outcomes in Children with Sickle Cell Anemia: A Single Center's Experience

Kristine Anne Karkoska, MD, Kevin E Todd, Jr., MD, Kelly Clapp, NP, Lynette Fenchel, NP, Theodosia A. Kalfa, MD PhD, Punam Malik, MD, Omar Niss, MD, Charles T. Quinn, MD, Russell E. Ware, MD PhD, Patrick T. McGann, MD MS

Blood (2020) 136 (Supplement 1): 34.

https://doi.org/10.1182/blood-2020-138754

Published: 2020

Journal Articles

FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease

R. Clark Brown, MD PhD, Kimberly Cruz, MD MS, Theodosia A. Kalfa, MD PhD, Frans A. Kuypers, PhD, Santosh L. Saraf, MD, Jeremie H. Estepp, MD, Luke R. Smart, MD, Punam Malik, MD, Mark Lerman, MD, Renae Mayer, MD, Maria D. Ribadeneira, PhD, Sanjeev Forsyth, MS, Patricia Schroeder, PhD, Eric Wu, PhD, Patrick Kelly, MD, Marilyn J. Telen, MD

Blood (2020) 136 (Supplement 1): 19–20.

https://doi.org/10.1182/blood-2020-134269

Published: 2020

Journal Articles

Early Results from a Phase 1/2 Study of Aru-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant

Michael Grimley, MD, Monika Asnani, PhD, Archana Shrestha, Sydney Felker, Carolyn Lutzko, PhD, Paritha I. Arumugam, PhD, Scott Witting, PhD, Jennifer Knight-Madden, MBBS, PhD, Omar Niss, MD, Charles T. Quinn, MD, Chris Lo, BS, Courtney R Little, BSN, Joseph W. McIntosh, MD, Punam Malik, MD

Blood (2020) 136 (Supplement 1): 20–21.

https://doi.org/10.1182/blood-2020-140963

Published: 2020

Journal Articles

Progression of Albuminuria in Sickle Cell Anemia: A Multicenter, Longitudinal Study

Omar Niss, MD, Adam Lane, PhD, Monika Asnani, PhD, Marianne McPherson Yee, MD MSc, Ashok Raj, Susan E Creary, MD, Courtney D. Fitzhugh, MD, Prasad V. Bodas, MD, Santosh L. Saraf, MD, Sharada Sarnaik, MD, Charles T. Quinn, MD, Prasad Devarajan, MD, Punam Malik, MD

Blood (2019) 134 (Supplement_1): 1004.

https://doi.org/10.1182/blood-2019-125860

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

Diminished Renal Pathology in a Mouse Model of Sickle Cell Anemia in Which Fibrinogen Binding to Mac-1 Is Inhibited

Md Nasimuzzaman, PhD, Paritha I. Arumugam, PhD, Eric S. Mullins, MD, Jeanne M James, MD, Katherine VandenHeuvel, MD, Marilou Narciso, BS, Maureen A. Shaw, MSc, Sarah McGraw, Bruce J. Aronow, PhD, Matthew J. Flick, PhD, Punam Malik, MD

Blood (2018) 132 (Supplement 1): 267.

https://doi.org/10.1182/blood-2018-99-112641

Published: 2018

Journal Articles

Clinical and Laboratory Benefits of Early Initiation of Hydroxyurea with Pharmacokinetic Guided Dosing for Young Children with Sickle Cell Anemia

Patrick T. McGann, MD, Omar Niss, MD, Min Dong, PhD, Anu Marahatta, PhD, Tomoyuki Mizuno, PhD, Karen Kalinyak, MD, Theodosia A. Kalfa, MD PhD, Punam Malik, MD, Charles T. Quinn, MD, Russell E. Ware, MD PhD, Alexander A. Vinks, PharmD, PhD FCP

Blood (2018) 132 (Supplement 1): 507.

https://doi.org/10.1182/blood-2018-99-112909

Published: 2018

Journal Articles

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

Blood (2018) 132 (Supplement 1): 1021.

https://doi.org/10.1182/blood-2018-99-119591

Published: 2018

Journal Articles

Diastolic Dysfunction Is Associated with Impaired Cardiopulmonary Exercise Performance in Sickle Cell Disease

Tarek Alsaied, MD MSc, Omar Niss, MD, Adam Powell, MD, Robert Fleck, MD, Punam Malik, MD, Clifford Chin, MD, Charles T. Quinn, MD MS, Michael D Taylor

Blood (2017) 130 (Supplement 1): 3547.

https://doi.org/10.1182/blood.V130.Suppl_1.3547.3547

Published: 2017

Journal Articles

Temporal Evolution of Diffuse Myocardial Fibrosis in Sickle Cell Anemia

Charles T. Quinn, MD MS, Omar Niss, MD, Robert Fleck, MD, Tarek Alsaied, MD MSc, Punam Malik, MD, Michael D. Taylor, MD

Blood (2017) 130 (Supplement 1): 3539.

https://doi.org/10.1182/blood.V130.Suppl_1.3539.3539

Published: 2017

Journal Articles

Personalized Hydroxyurea Dosing to Reduce Time to MTD and Optimize the HbF Response: Results from the TREAT Study

Patrick T. McGann, MD MS, Omar Niss, MD, Min Dong, PhD, Anu Marahatta, PhD, Kathryn L McElhinney, BS, Thad A. Howard, MS, Tomoyuki Mizuno, PhD, Theodosia A. Kalfa, MD PhD, Karen Kalinyak, MD, Punam Malik, MD, Charles T. Quinn, MD MS, Russell E. Ware, MD PhD, Alexander A. Vinks, PharmD, PhD FCP

Blood (2017) 130 (Supplement 1): 615.

https://doi.org/10.1182/blood.V130.Suppl_1.615.615

Published: 2017

Journal Articles

A Precise Tool to Edit Human Hematopoietic Stem Cells at High Efficiency and Decipher and Optimize the Different Double Strand Break Repair Outcomes

Rajeswari Jayavaradhan, MS, Devin Pillis, MS, Sarah McGraw, Loberg Anastacia, Paul R. Andreassen, PhD, Punam Malik, MD

Blood (2017) 130 (Supplement 1): 2067.

https://doi.org/10.1182/blood.V130.Suppl_1.2067.2067

Published: 2017

Journal Articles

Methacholine Challenge Test Reveals a High Prevalence of Subclinical Severe Airway Hyper-Reactivity in Children and Yound Adults with Sickle Cell Disease Which Correlates with Higher Sickle Acute Events and Acute Chest

Marthe-Sandrine Eiymo Mwa Mpollo, PhD, Sharat Chandra, MBBS,MD, Michael R. DeBaun, MD MPH, Uwe Christians, MD PhD, William Hardie, MD, Karen Kalinyak, MD, Punam Malik, MD

Blood (2017) 130 (Supplement 1): 761.

https://doi.org/10.1182/blood.V130.Suppl_1.761.761

Published: 2017

Journal Articles

Uncoupling Sickle Hematopoiesis Induced Angiotensin Receptor Signaling in Sickle Nephropathy

Swarnava Roy, PhD, Parul Rai, MD, Marthe-Sandrine Eiymo Mwa Mpollo, PhD, Kyung-Hee Chang, PhD, Tilat Rizvi, Loberg Anastacia, Bruce J. Aronow, PhD, Tadashi Inagami, PhD, David Witte, MD, Jose A Cancelas, MD PhD, Punam Malik, MD

Blood (2017) 130 (Supplement 1): 440.

https://doi.org/10.1182/blood.V130.Suppl_1.440.440

Published: 2017

Journal Articles

Foamy Virus Backbone Has Insulator Properties Which Remarkably Reduce Its Genotoxicity Potential

Michael Goodman, MD, Paritha Arumugam, PhD, Devin Pillis, MS, Danielle Lynn, MS, Johannes C.M. Van der Loo, PhD, Mehdi Keddache, PhD, Thomas R. Bauer, Jr., PhD, Dennis D. Hickstein, MD, David W Russell, MD PhD, Punam Malik, MD

Blood (2016) 128 (22): 1002.

https://doi.org/10.1182/blood.V128.22.1002.1002

Published: 2016

Journal Articles

Individualized Dosing of Hydroxyurea for Children with Sickle Cell Anemia Using a Population Pharmacokinetic-Based Model: The TREAT Study

Patrick T. McGann, MD MS, Min Dong, PhD, Anu Marahatta, PhD, Thad A. Howard, MS, Tomoyuki Mizuno, PhD, Karen A. Kalinyak, MD, Theodosia A. Kalfa, MD PhD, Punam Malik, MD, Omar Niss, MD, Charles T Quinn, MD, Russell E. Ware, MD PhD, Alexander A. Vinks, PharmD, PhD

Blood (2016) 128 (22): 3652.

https://doi.org/10.1182/blood.V128.22.3652.3652

Published: 2016

NARROW

Bone Marrow (BM) Delivery of Genetically-Modified (gm) Adult CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Improves Homing and Engraftment of Short-Term Progenitors over Long-Term Repopulating Hematopoietic Stem Cells

Rapid and Automated Quantitation of Dense Red Blood Cells: A Robust Biomarker of Therapeutic Response to Early Initiation of Hydroxyurea in Young Children with Sickle Cell Anemia

Oral Administration of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Has Potent Anti-Sickling Effects in a Sickle Cell Anemia (SCA) Mouse Model, Resulting in Improved RBC Survival and Hemoglobin Levels

Angiotensin Signaling Is Essential for Stress Erythropoiesis but Results in Retention of Dysfunctional Mitochondria in Erythrocytes That Generate Excessive Reactive Oxygen Species

Increased Hydroxyurea Prescribing Practices over Ten Years with Improved Clinical Outcomes in Children with Sickle Cell Anemia: A Single Center's Experience

FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease

Early Results from a Phase 1/2 Study of Aru-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant

Progression of Albuminuria in Sickle Cell Anemia: A Multicenter, Longitudinal Study

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

Diminished Renal Pathology in a Mouse Model of Sickle Cell Anemia in Which Fibrinogen Binding to Mac-1 Is Inhibited

Clinical and Laboratory Benefits of Early Initiation of Hydroxyurea with Pharmacokinetic Guided Dosing for Young Children with Sickle Cell Anemia

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

Diastolic Dysfunction Is Associated with Impaired Cardiopulmonary Exercise Performance in Sickle Cell Disease

Temporal Evolution of Diffuse Myocardial Fibrosis in Sickle Cell Anemia

Personalized Hydroxyurea Dosing to Reduce Time to MTD and Optimize the HbF Response: Results from the TREAT Study

A Precise Tool to Edit Human Hematopoietic Stem Cells at High Efficiency and Decipher and Optimize the Different Double Strand Break Repair Outcomes

Methacholine Challenge Test Reveals a High Prevalence of Subclinical Severe Airway Hyper-Reactivity in Children and Yound Adults with Sickle Cell Disease Which Correlates with Higher Sickle Acute Events and Acute Chest

Uncoupling Sickle Hematopoiesis Induced Angiotensin Receptor Signaling in Sickle Nephropathy

Foamy Virus Backbone Has Insulator Properties Which Remarkably Reduce Its Genotoxicity Potential

Individualized Dosing of Hydroxyurea for Children with Sickle Cell Anemia Using a Population Pharmacokinetic-Based Model: The TREAT Study

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American Society of Hematology

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American Society of Hematology

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