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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

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

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

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

Journal Articles

Knowledge, Cultural, and Structural Barriers to Thalassemia Screening in Migrant Populations in Thailand

Julia Z. Xu, MD MScGH, Wilaslak Tanongsaksakul, MD, Thidarat Suksangpleng, PhD, Supachai Ekwattanakit, PhD MD, Suchada Riolueang, MSc, Bonnie N. Kaiser, PhD, Nathan M. Thielman, MD, Marilyn J. Telen, MD, Vip Viprakasit, MD DPhil

Blood (2018) 132 (Supplement 1): 2228.

https://doi.org/10.1182/blood-2018-99-114117

Published: 2018

Journal Articles

RNA-Sequencing of Isolated Cell Populations Expressing Human APOL1 Risk Variants Reveals Molecular Correlates of Sickle Cell Nephropathy in Zebrafish Podocytes

Joseph L Bundy, PhD BS, Blair R Anderson, PhD, Ludmila Francescatto, PhD, Melanie E Garrett, MS, Karen L Soldano, BS, Marilyn J. Telen, MD, Erica E Davis, PhD, Allison E Ashley-Koch, PhD

Blood (2017) 130 (Supplement 1): 2236.

https://doi.org/10.1182/blood.V130.Suppl_1.2236.2236

Published: 2017

Journal Articles

Thrombospondin-1 Polymorphisms Are Associated with Chronic Kidney Disease in Sickle Cell Anemia

Seethal A. Jacob, MD, Enrico M. Novelli, MD MS, Jeffrey S. Isenberg, MD MPH, Melanie E. Garrett, MS, Yanxia Chu, MD, Karen L. Soldano, BS, Kenneth I. Ataga, MD, Marilyn J. Telen, MD, Allison E. Ashley-Koch, PhD, Yingze Zhang, PhD, Gregory J Kato, MD

Blood (2016) 128 (22): 2491.

https://doi.org/10.1182/blood.V128.22.2491.2491

Published: 2016

Journal Articles

Effect of FXIII Polymorphism on Formation of Heterocellular Aggregates in SCD

Jennifer Minjia Chang, Martha Delahunty, PhD, Milena Batchvarova, MS, Karen L. Soldano, BS, J. Michael Cook, PhD, Marilyn J. Telen, MD

Blood (2016) 128 (22): 3648.

https://doi.org/10.1182/blood.V128.22.3648.3648

Published: 2016

Journal Articles

Use of Mobile Technology to Monitor Pain and Reduce Outpatient, Emergency Department (ED), and Hospital Visits for Sickle Cell Pain Crisis

Kalindi Yajnik Narine, MD, Jennifer Minjia Chang, Jude Jonassaint, RN, Marilyn J. Telen, MD, Nirmish Shah, MD

Blood (2016) 128 (22): 2390.

https://doi.org/10.1182/blood.V128.22.2390.2390

Published: 2016

Journal Articles

Factors Related to the Progression of Sickle Cell Disease Nephropathy

Julia Z. Xu, MD, Melanie E. Garrett, MS, Karen L. Soldano, BS, Sean T. Chen, Allison E. Ashley-Koch, PhD, Marilyn J. Telen, MD

Blood (2016) 128 (22): 9.

https://doi.org/10.1182/blood.V128.22.9.9

Published: 2016

Journal Articles

GWAS Meta-Analysis of Glomerular Filtration Rate in Three Cohorts of Sickle Cell Disease Patients and In Vivo Functional Analysis Reveals Potential Nephropathy Candidate Genes

Blair R. Anderson, BS, Melanie E. Garrett, MS, Karen L. Soldano, BS, Eugene P. Orringer, MD, James R. Eckman, MD, Ludmila Francescatto, PhD, Erica E. Davis, PhD, Mark T. Gladwin, MD, Victor R. Gordeuk, MD, Marilyn J. Telen, MD, Allison E. Ashley-Koch, PhD

Blood (2016) 128 (22): 269.

https://doi.org/10.1182/blood.V128.22.269.269

Published: 2016

Journal Articles

Beyond hydroxyurea: new and old drugs in the pipeline for sickle cell disease

Marilyn J. Telen

Blood (2016) 127 (7): 810–819.

https://doi.org/10.1182/blood-2015-09-618553

Published: 2016

NARROW

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

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

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

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

Knowledge, Cultural, and Structural Barriers to Thalassemia Screening in Migrant Populations in Thailand

RNA-Sequencing of Isolated Cell Populations Expressing Human APOL1 Risk Variants Reveals Molecular Correlates of Sickle Cell Nephropathy in Zebrafish Podocytes

Thrombospondin-1 Polymorphisms Are Associated with Chronic Kidney Disease in Sickle Cell Anemia

Effect of FXIII Polymorphism on Formation of Heterocellular Aggregates in SCD

Use of Mobile Technology to Monitor Pain and Reduce Outpatient, Emergency Department (ED), and Hospital Visits for Sickle Cell Pain Crisis

Factors Related to the Progression of Sickle Cell Disease Nephropathy

GWAS Meta-Analysis of Glomerular Filtration Rate in Three Cohorts of Sickle Cell Disease Patients and In Vivo Functional Analysis Reveals Potential Nephropathy Candidate Genes

Beyond hydroxyurea: new and old drugs in the pipeline for sickle cell disease

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