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Journal Articles

Splenic Ossification in Sickle Cell Anemia Mice

Mengna Chi, Katherine Vandenheuvel, Punam Malik

Blood (2024) 144 (Supplement 1): 5283.

https://doi.org/10.1182/blood-2024-209046

Published: 2024

Journal Articles

A Novel Mouse Model of Hemoglobin-SC Disease: Bridging the Knowledge Gap in Sickle Cell Disease

Tahereh Setayesh, Mengna Chi, Zachery Oestreicher, Masahide Sakabe, Xin Mei, Katherine Vandenheuvel, Tim M. Townes, Yueh-Chiang Hu, Punam Malik

Blood (2024) 144 (Supplement 1): 620.

https://doi.org/10.1182/blood-2024-206241

Published: 2024

Journal Articles

Underutilization of Disease-Modifying Therapies in Sickle Cell Disease: A Real-World Analysis from the ASH Research Collaborative Data Hub

Omar Niss, Matthew Fenchel, Erin Kingsley, Teresa S. Latham, Punam Malik, Charles T. Quinn

Blood (2024) 144 (Supplement 1): 2312.

https://doi.org/10.1182/blood-2024-206015

Published: 2024

Journal Articles

Longitudinal Assessment of Myocardial Fibrosis in Sickle Cell Disease

Omar Niss, Cara Morin, Sean M Lang, Tarek Alsaied, MacKenzie Tasset, Punam Malik, Charles T. Quinn

Blood (2024) 144 (Supplement 1): 799.

https://doi.org/10.1182/blood-2024-200951

Published: 2024

Journal Articles

Blockade of Angiotensin II Signaling Ameliorates Cardiac Fibrosis and Improves Diastolic Dysfunction in Mice with Sickle Cell Anemia

Keara Little, Zhenqi Zhu, Oluwabukola Gbotosho, Punam Malik

Blood (2024) 144 (Supplement 1): 2480.

https://doi.org/10.1182/blood-2024-208670

Published: 2024

Journal Articles

CRISPR/CAS9 Gene Editing of MIR155HG in Primary Human T Cells to Prevent Acute Graft-Versus-Host Disease

Shrijan Khanal, Lotus Neidemire-Colley, Kara M Braunreiter, Katiri Snyder, Yandi Gao, Sonu Kalyan, Meisam Naeimi Kararoudi, Molly Erin Duszynski, Mengna Chi, Punam Malik, Hannah Choe, Ramiro Garzon, Parvathi Ranganathan

Blood (2022) 140 (Supplement 1): 10200–10201.

https://doi.org/10.1182/blood-2022-159599

Published: 2022

Journal Articles

Early initiation of disease-modifying therapy can impede or prevent diffuse myocardial fibrosis in sickle cell anemia

Omar Niss, Jon Detterich, John C. Wood, Thomas D. Coates, Punam Malik, Michael D. Taylor, Charles T. Quinn

Blood (2022) 140 (11): 1322–1324.

https://doi.org/10.1182/blood.2021015303

Published: 2022

Includes: Supplemental data

Journal Articles

Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning

Michael Grimley, Monika Asnani, Archana Shrestha, Sydney Felker, Carolyn Lutzko, Paritha I. Arumugam, Scott Witting, Jennifer Knight-Madden, Omar Niss, Charles T. Quinn, Chris Lo, Courtney R. Little, Alisa Dong, Punam Malik

Blood (2021) 138 (Supplement 1): 3970.

https://doi.org/10.1182/blood-2021-147469

Published: 2021

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

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

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

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

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

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

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

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

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

NARROW

Splenic Ossification in Sickle Cell Anemia Mice

A Novel Mouse Model of Hemoglobin-SC Disease: Bridging the Knowledge Gap in Sickle Cell Disease

Underutilization of Disease-Modifying Therapies in Sickle Cell Disease: A Real-World Analysis from the ASH Research Collaborative Data Hub

Longitudinal Assessment of Myocardial Fibrosis in Sickle Cell Disease

Blockade of Angiotensin II Signaling Ameliorates Cardiac Fibrosis and Improves Diastolic Dysfunction in Mice with Sickle Cell Anemia

CRISPR/CAS9 Gene Editing of MIR155HG in Primary Human T Cells to Prevent Acute Graft-Versus-Host Disease

Early initiation of disease-modifying therapy can impede or prevent diffuse myocardial fibrosis in sickle cell anemia

Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning

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

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

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

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

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

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

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

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

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

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American Society of Hematology

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American Society of Hematology

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