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Journal Articles

Molecular Mechanism Underlying Excessive Phosphatidylserine Exposure in Sickle Red Blood Cells and Potential Therapeutics

Pengfei Liang, Ke Zoe Shan, Ryan Chou, Yui Chun Serena Wan, Martha Delahunty, Sanjay Khandelwal, Samuel Francis, Gow Arepally, Marilyn J. Telen, Huanghe Yang

Blood (2024) 144 (Supplement 1): 174.

https://doi.org/10.1182/blood-2024-199340

Published: 2024

Journal Articles

Novel Loci Associated with Acute Chest Syndrome in Sickle Cell Disease Patients

Anvita Kulshrestha, Melanie E. Garrett, Marilyn J. Telen, Allison E. Ashley-Koch

Blood (2024) 144 (Supplement 1): 1110.

https://doi.org/10.1182/blood-2024-206550

Published: 2024

Journal Articles

Electronic Heath Record Phenotypes to Classify Sickle Cell Anemia Versus Other Subtypes of Sickle Cell Disease

John J Strouse, Mariam Kayle, Charity I Oyedeji, Jennifer A. Rothman, Nathan Bihlmeyer, Allison E. Ashley-Koch, Melanie E. Garrett, Marilyn J. Telen

Blood (2024) 144 (Supplement 1): 7652.

https://doi.org/10.1182/blood-2024-200373

Published: 2024

Journal Articles

Etavopivat Reduces Incidence of Vaso-Occlusive Crises in Patients with Sickle Cell Disease: HIBISCUS Trial Phase 2 Results through 52 Weeks

Sophia Delicou, Fuad El Rassi, Biree Andemariam, Miguel R. Abboud, Julie Kanter, Marilyn J. Telen, Jessie Githanga, Adlette Inati, Ibrahim Idris, Sunil Navani, Eric Wu, Andrew Eisenberger

Blood (2024) 144 (Supplement 1): 179.

https://doi.org/10.1182/blood-2024-204962

Published: 2024

Journal Articles

Nontargeted Plasma Proteomic Analysis in Sickle Cell Disease Implicates Pathways Involving Cell Structure, Immunity and Heme and Oxygen Transport in the Occurrence of Leg Ulcers

Melanie E. Garrett, Matthew W. Foster, Martha Delahunty, Gregory J. Kato, Caterina P. Minniti, Allison E. Ashley-Koch, Marilyn J. Telen

Blood (2024) 144 (Supplement 1): 5288.

https://doi.org/10.1182/blood-2024-210971

Published: 2024

Journal Articles

Evaluation of the Role of PIEZO1 G enetic Variants in Red Blood Cell Health in Sickle Cell Disease

Mandisa L Keswa, Melanie E. Garrett, Marilyn J. Telen, Allison E. Ashley-Koch

Blood (2024) 144 (Supplement 1): 5276.

https://doi.org/10.1182/blood-2024-207732

Published: 2024

Journal Articles

Red Cell Exchange Decreases Sickle RBC-Induced Release of MPO from Neutrophils

Y-Nhi Bui, Kimberly Boyle, Milena Batchvarova, Martha Delahunty, Marilyn J. Telen, Grace M Lee

Blood (2024) 144 (Supplement 1): 5277.

https://doi.org/10.1182/blood-2024-207819

Published: 2024

Journal Articles

A Phase 2 Prospective Randomized Trial of Topical Sodium Nitrite in Patients with Sickle Cell Disease and Leg Ulcers

Caterina P. Minniti, Gregory J. Kato, Melanie E. Garrett, Allison E. Ashley-Koch, Marilyn J. Telen

Blood (2024) 144 (Supplement 1): 2512.

https://doi.org/10.1182/blood-2024-199793

Published: 2024

Journal Articles

Deciphering and disrupting PIEZO1-TMEM16F interplay in hereditary xerocytosis

Pengfei Liang, Yang Zhang, Yui Chun S. Wan, Shang Ma, Ping Dong, Augustus J. Lowry, Samuel J. Francis, Sanjay Khandelwal, Martha Delahunty, Marilyn J. Telen, John J. Strouse, Gowthami M. Arepally, Huanghe Yang

Blood (2024) 143 (4): 357–369.

https://doi.org/10.1182/blood.2023021465

Published: 2024

Includes: Multimedia, Supplemental data

Journal Articles

PIEZO1-TMEM16F Interplay in Hereditary Xerocytosis

Pengfei Liang, Yang Zhang, Yui Chun Serena Wan, Shang Ma, Ping Dong, Augustus Lowry, Samuel Francis, Sanjay Khandelwal, Martha Delahunty, Marilyn J. Telen, John J Strouse, Gowthami Arepally, Huanghe Yang

Blood (2023) 142 (Supplement 1): 3818.

https://doi.org/10.1182/blood-2023-178911

Published: 2023

Journal Articles

Biologic Assessment of RBC Biology and Neutrophil Activation: Correlation with Sickle Cell Disease Activity

Marilyn J. Telen, Milena Batchvarova, Martha Delahunty

Blood (2023) 142 (Supplement 1): 1130.

https://doi.org/10.1182/blood-2023-187032

Published: 2023

Journal Articles

Etavopivat, an Allosteric Activator of Pyruvate Kinase-R, Improves Sickle RBC Functional Health and Survival and Reduces Systemic Markers of Inflammation and Hypercoagulability in Patients with Sickle Cell Disease: An Analysis of Exploratory Studies in a Phase 1 Study

Theodosia A. Kalfa, Marilyn J. Telen, Santosh L. Saraf, R. Clark Clark Brown, Katie Giger Seu, Sandra K. Larkin, Maria D. Ribadeneira, Patricia Schroeder, Eric Wu, Patrick Kelly, Frans A. Kuypers

Blood (2021) 138 (Supplement 1): 8.

https://doi.org/10.1182/blood-2021-147078

Published: 2021

Journal Articles

Activation of Pyruvate Kinase-R with Etavopivat (FT-4202) Is Well Tolerated, Improves Anemia, and Decreases Intravascular Hemolysis in Patients with Sickle Cell Disease Treated for up to 12 Weeks

R. Clark Clark Brown, Santosh L. Saraf, Kimberly Cruz, Modupe Idowu, Theodosia A. Kalfa, James Geib, Sanjeev Forsyth, Patricia Schroeder, Eric Wu, Patrick Kelly, Marilyn J. Telen

Blood (2021) 138 (Supplement 1): 9.

https://doi.org/10.1182/blood-2021-147091

Published: 2021

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 Initiation of Treatment with Rivipansel for Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves Earlier Discontinuation of IV Opioids and Shorter Hospital Stay: Reset Clinical Trial Analysis

Carlton D. Dampier, MD, Marilyn J. Telen, MD, Ted Wun, MD, Wally R Smith, MD, R. Clark Brown, MD PhD, Payal Desai, MD, Fuad A El Rassi, MD, Julie Kanter, MD, Beng R. Fuh, MD, Yves D. Pastore, MD, Jennifer A. Rothman, MD, James G. Taylor, MD, David Readett, BM, BS, MRCP, Jay N Lozier, MDPhD, John L. Magnani, PhD, Helen M Thackray, MD, Kathryn L. Hassell, MD

Blood (2020) 136 (Supplement 1): 18–19.

https://doi.org/10.1182/blood-2020-134803

Published: 2020

Journal Articles

Genome Wide Association Analysis of Iron Overload in the Trans-Omics for Precision Medicine (TOPMed) Sickle Cell Disease Cohorts

Blood (2020) 136 (Supplement 1): 52.

https://doi.org/10.1182/blood-2020-142809

Published: 2020

Journal Articles

An Adaptive, Randomized, Placebo-Controlled, Double-Blind, Multi-Center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE)

Kenneth W. Wood, PhD, James Geib, BS, Eric Wu, PhD, Jason Berlin, MS, Irena Webster, MPH, MA, Kenneth I. Ataga, MD, Jo Howard, MBBChir, MRCP, FRCPath, Jeremie H. Estepp, MD, Marilyn J. Telen, MD, Julie Brevard, MPH

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

https://doi.org/10.1182/blood-2020-134266

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

Identification of Optimal Thalassemia Screening Strategies for Migrant Populations in Thailand: A Mixed-Methods Approach

Julia Z. Xu, MD MSc, Meghan Foe, Wilaslak Tanongsaksakul, MD, Thidarat Suksangpleng, PhD, Supachai Ekwattanakit, PhDMD, Suchada Riolueang, MSc, Marilyn J. Telen, MD, Bonnie N. Kaiser, PhD, Vip Viprakasit, MD DPhil

Blood (2019) 134 (Supplement_1): 2112.

https://doi.org/10.1182/blood-2019-131805

Published: 2019

Journal Articles

Mechanism Underlying a Role for Factor XIII (FXIII) Polymorphism in Sickle Cell Disease-Associated Priapism

Marilyn J. Telen, MD, Milena Batchvarova, MS, Joan D Beckman, MD PhD, Martha Delahunty, PhD, Karen L Soldano, BS, Allison E Ashley-Koch, PhD, Alisa S. Wolberg, PhD

Blood (2018) 132 (Supplement 1): 2361.

https://doi.org/10.1182/blood-2018-99-116946

Published: 2018

NARROW

Molecular Mechanism Underlying Excessive Phosphatidylserine Exposure in Sickle Red Blood Cells and Potential Therapeutics

Novel Loci Associated with Acute Chest Syndrome in Sickle Cell Disease Patients

Electronic Heath Record Phenotypes to Classify Sickle Cell Anemia Versus Other Subtypes of Sickle Cell Disease

Etavopivat Reduces Incidence of Vaso-Occlusive Crises in Patients with Sickle Cell Disease: HIBISCUS Trial Phase 2 Results through 52 Weeks

Nontargeted Plasma Proteomic Analysis in Sickle Cell Disease Implicates Pathways Involving Cell Structure, Immunity and Heme and Oxygen Transport in the Occurrence of Leg Ulcers

Evaluation of the Role of PIEZO1 G enetic Variants in Red Blood Cell Health in Sickle Cell Disease

Red Cell Exchange Decreases Sickle RBC-Induced Release of MPO from Neutrophils

A Phase 2 Prospective Randomized Trial of Topical Sodium Nitrite in Patients with Sickle Cell Disease and Leg Ulcers

Deciphering and disrupting PIEZO1-TMEM16F interplay in hereditary xerocytosis

PIEZO1-TMEM16F Interplay in Hereditary Xerocytosis

Biologic Assessment of RBC Biology and Neutrophil Activation: Correlation with Sickle Cell Disease Activity

Etavopivat, an Allosteric Activator of Pyruvate Kinase-R, Improves Sickle RBC Functional Health and Survival and Reduces Systemic Markers of Inflammation and Hypercoagulability in Patients with Sickle Cell Disease: An Analysis of Exploratory Studies in a Phase 1 Study

Activation of Pyruvate Kinase-R with Etavopivat (FT-4202) Is Well Tolerated, Improves Anemia, and Decreases Intravascular Hemolysis in Patients with Sickle Cell Disease Treated for up to 12 Weeks

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 Initiation of Treatment with Rivipansel for Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves Earlier Discontinuation of IV Opioids and Shorter Hospital Stay: Reset Clinical Trial Analysis

Genome Wide Association Analysis of Iron Overload in the Trans-Omics for Precision Medicine (TOPMed) Sickle Cell Disease Cohorts

An Adaptive, Randomized, Placebo-Controlled, Double-Blind, Multi-Center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE)

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

Identification of Optimal Thalassemia Screening Strategies for Migrant Populations in Thailand: A Mixed-Methods Approach

Mechanism Underlying a Role for Factor XIII (FXIII) Polymorphism in Sickle Cell Disease-Associated Priapism

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American Society of Hematology

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