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Journal Articles

Initial Results in a Phase 1b Trial of PB-04 in Sickle Cell Disease Demonstrate Fetal Hemoglobin Induction, Additive Activity with Hydroxyurea, and Improved Red Blood Cell Sickling Parameters

Kevin H.M. Kuo, Sylvia Titi Singer, Gershwin Theophilus Blyden, Lanetta Bronté-Hall, Aidan Faller, Abdullah Kutlar, Frans A. Kuypers, Aliya U. Zaidi, Patrick C. Hines, Niren Patel, Sandra Larkin, Hanny Al-Samkari, Sujit Sheth, Seyed Mehdi Nouraie, Susan Perrine

Blood (2023) 142 (Supplement 1): 1148.

https://doi.org/10.1182/blood-2023-184627

Published: 2023

Journal Articles

Roxyscan: A Novel Method to Assess Red Blood Cell Resilience to Oxidative Stress in Sickle Cell Disease

Carolina A. Hernandez, Marissa J.M. Traets, Myrthe J. van Dijk, Wouter W. van Solinge, Frans A. Kuypers, Minke A.E. Rab, Eduard J. Van Beers, Richard van Wijk

Blood (2023) 142 (Supplement 1): 2497.

https://doi.org/10.1182/blood-2023-188676

Published: 2023

Journal Articles

The Effect of Voxelotor on Exercise Capacity of Youths with Sickle Cell Anemia

Vivian Phan, Laura Caldarera, Ana Lucia Cortez, Kari Wheeler, Sandra K. Larkin, Sangah Nancy Park, Robin Yates Dulman, Noravy Briere, Angela Lewis, Frans A. Kuypers, Jared Hershenson, Elizabeth Yang

Blood (2021) 138 (Supplement 1): 2045.

https://doi.org/10.1182/blood-2021-149163

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

Differential Acetone Extraction of Total and Hemoprotein-Unbound Heme to Quantify Heme Binding Capacity of Plasma in Patients with Sickle Cell Disease: The Role of Heme Scavengers

Madhav Vissa, Eric Soupene, PhD, Sandra K. Larkin, MS, Lynne D. Neumayr, MD, Elliott P. Vichinsky, MD, Frans A. Kuypers, PhD

Blood (2020) 136 (Supplement 1): 15.

https://doi.org/10.1182/blood-2020-134717

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

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Julie Kanter, MD, Mark C. Walters, MD, Matthew M. Hsieh, MD, Lakshmanan Krishnamurti, MD, Janet Kwiatkowski, MD, Rammurti T Kamble, MD, Christof von Kalle, MD, Frans A. Kuypers, PhD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Marcelyne Joseney-Antoine, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH, John F. Tisdale, MD

Blood (2016) 128 (22): 1176.

https://doi.org/10.1182/blood.V128.22.1176.1176

Published: 2016

Journal Articles

Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease

Julie Kanter, MD, Mark C. Walters, MD, Matthew Hsieh, MD, Alexis A. Thompson, MD, Lakshmanan Krishnamurti, MD, Janet Kwiatkowski, MD, Rammurti T Kamble, MD, Christof von Kalle, MD, Frans A. Kuypers, PhD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Laura Sandler, MPH, Sandeep Soni, MD, John F. Tisdale, MD

Blood (2015) 126 (23): 3233.

https://doi.org/10.1182/blood.V126.23.3233.3233

Published: 2015

Journal Articles

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

Blood (2015) 126 (23): 202.

https://doi.org/10.1182/blood.V126.23.202.202

Published: 2015

Journal Articles

Simvastatin Reduces Frequency of Vaso-Occlusive Pain in Sickle Cell Disease: A Pilot Efficacy Trial

Carolyn C. Hoppe, MD, Eufemia Jacob, PhD RN, Frans A. Kuypers, PhD, Lori Styles, MD

Blood (2015) 126 (23): 545.

https://doi.org/10.1182/blood.V126.23.545.545

Published: 2015

Journal Articles

Pan-Selectin Antagonist Rivipansel (GMI-1070) Reduces Soluble E-Selectin Levels While Improving Clinical Outcomes in SCD Vaso-Occlusive Crisis

Ted Wun, MD, Marilyn J. Telen, MD, Lakshmanan Krishnamurti, MD, Timothy L. McCavit, MD, Laura M DeCastro, MD, Henry Flanner, ME, Frans A. Kuypers, PhD, Sandra K. Larkin, MS, Seungshin Rhee, MS, John L. Magnani, PhD, Helen M. Thackray, MD

Blood (2014) 124 (21): 2704.

https://doi.org/10.1182/blood.V124.21.2704.2704

Published: 2014

Journal Articles

Microparticles As Biomarkers Of Osteonecrosis Of The Hip In Sickle Cell Disease

Anne M Marsh, MD, Raymond Schiffelers, PhD, Ginny Gildengorin, PhD, Frans A Kuypers, PhD, Carolyn Hoppe, MD

Blood (2013) 122 (21): 2226.

https://doi.org/10.1182/blood.V122.21.2226.2226

Published: 2013

Journal Articles

Citrate Synthase Activity Is Increased In Children With Sickle Cell Disease (SCD) On Hydroxyurea (HU) Therapy

Claudia R. Morris, MD, Cornelia Latronica, MD, Frans A Kuypers, PhD, Howard Rosenfeld, MD, Sandra K Larkin, MS, Stephanie Doniger, MD, Elliott Vichinsky, MD, Sruti Shiva, PhD

Blood (2013) 122 (21): 4690.

https://doi.org/10.1182/blood.V122.21.4690.4690

Published: 2013

Journal Articles

Sildenafil Therapy in Patients with Thalassemia and an Elevated Tricuspid Regurgitant Jet Velocity (TRV) On Doppler Echocardiography At Risk for Pulmonary Hypertension: Report From the Thalassemia Clinical Research Network

Claudia R. Morris, MD, Hae-Young Kim, DrPH, John C Wood, MD, Felicia Trachtenberg, PhD, Elizabeth S Klings, MD, John B. Porter, MD, Nancy Sweeters, RN, PNP, Nancy F Olivieri, MD, Janet L. Kwiatkowski, MD, Sylvia Titi Singer, MD, Ali T Taher, MD, Ellis J. Neufeld, MD, PhD, Alexis A. Thompson, MD, MPH, Vandana Sachdev, M.D., Sandra K Larkin, MS, Jung H Suh, PhD, Frans A Kuypers, PhD, Elliott Vichinsky, MD

Blood (2012) 120 (21): 1023.

https://doi.org/10.1182/blood.V120.21.1023.1023

Published: 2012

Journal Articles

Tricuspid Regurgitant Jet Velocity (TRV), Biomarkers of Hemolysis, and Impact of Oxygen Therapy in Children with Sickle Cell Disease (SCD) and Vaso-Occlusive Pain Episodes (VOE)

Cornelia Latronica, MD, Frans A Kuypers, PhD, Howard Rosenfeld, MD, Sandra K Larkin, MS, Stephanie Doniger, MD, Elliott Vichinsky, MD, Claudia R. Morris, MD

Blood (2012) 120 (21): 4752.

https://doi.org/10.1182/blood.V120.21.4752.4752

Published: 2012

Journal Articles

Treatment with a Novel Dinitroazetidine, Abdnaz, Improves Nitrite Reductase Activity of Sickle Red Blood Cells

Marcel H.A.M. Fens, PhD, Sandra K Larkin, MS, William L Fitch, PhD, Jan Scicinski, PhD, Bryan Oronsky, MD, Frans A Kuypers, PhD

Blood (2012) 120 (21): 3246.

https://doi.org/10.1182/blood.V120.21.3246.3246

Published: 2012

Journal Articles

Cardiopulmonary and Laboratory Profiling of Patients with Thalassemia At Risk for Pulmonary Hypertension: Report From the Thalassemia Clinical Research Network.

Claudia R. Morris, MD, Hae-Young Kim, DrPH, John C Wood, MD, Ph.D, Felicia Trachtenberg, PhD, Elizabeth S Klings, MD, John B. Porter, MD, Nancy Sweeters, RN, PNP, Nancy F Olivieri, MD, Janet L. Kwiatkowski, MD, Sylvia Titi Singer, MD, Ali T Taher, MD, Ellis J. Neufeld, MD, PhD, Alexis A. Thompson, MD, MPH, Sandra K Larkin, MS, Jung H Suh, PhD, Elliott Vichinsky, MD, Frans A Kuypers, PhD

Blood (2012) 120 (21): 2122.

https://doi.org/10.1182/blood.V120.21.2122.2122

Published: 2012

Journal Articles

Pan-Selectin Antagonist GMI-1070 Affects Biomarkers of Adhesion, Activation and the Coagulation Cascade in Sickle Cell Adults At Steady State

John L. Magnani, PhD, Frans A Kuypers, PhD, John T. Patton, PhD, Sandra K Larkin, MS, Lori Styles, MD, Laura M DeCastro, MD, Marilyn J. Telen, MD, Ted Wun, MD, Helen Thackray, MD

Blood (2012) 120 (21): 87.

https://doi.org/10.1182/blood.V120.21.87.87

Published: 2012

Journal Articles

NO or No NO, Increased Reduction of Nitrite to Nitric Oxide by Modified Red Blood Cells

Marcel H.A.M. Fens, PhD, Sandra K Larkin, Claudia R. Morris, MD, Bill Fitch, PhD, Jan Scicinski, PhD, Bryan Oronsky, MD, Frans A Kuypers, PhD

Blood (2011) 118 (21): 2125.

https://doi.org/10.1182/blood.V118.21.2125.2125

Published: 2011

NARROW

Initial Results in a Phase 1b Trial of PB-04 in Sickle Cell Disease Demonstrate Fetal Hemoglobin Induction, Additive Activity with Hydroxyurea, and Improved Red Blood Cell Sickling Parameters

Roxyscan: A Novel Method to Assess Red Blood Cell Resilience to Oxidative Stress in Sickle Cell Disease

The Effect of Voxelotor on Exercise Capacity of Youths with Sickle Cell Anemia

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

Differential Acetone Extraction of Total and Hemoprotein-Unbound Heme to Quantify Heme Binding Capacity of Plasma in Patients with Sickle Cell Disease: The Role of Heme Scavengers

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

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

Simvastatin Reduces Frequency of Vaso-Occlusive Pain in Sickle Cell Disease: A Pilot Efficacy Trial

Pan-Selectin Antagonist Rivipansel (GMI-1070) Reduces Soluble E-Selectin Levels While Improving Clinical Outcomes in SCD Vaso-Occlusive Crisis

Microparticles As Biomarkers Of Osteonecrosis Of The Hip In Sickle Cell Disease

Citrate Synthase Activity Is Increased In Children With Sickle Cell Disease (SCD) On Hydroxyurea (HU) Therapy

Sildenafil Therapy in Patients with Thalassemia and an Elevated Tricuspid Regurgitant Jet Velocity (TRV) On Doppler Echocardiography At Risk for Pulmonary Hypertension: Report From the Thalassemia Clinical Research Network

Tricuspid Regurgitant Jet Velocity (TRV), Biomarkers of Hemolysis, and Impact of Oxygen Therapy in Children with Sickle Cell Disease (SCD) and Vaso-Occlusive Pain Episodes (VOE)

Treatment with a Novel Dinitroazetidine, Abdnaz, Improves Nitrite Reductase Activity of Sickle Red Blood Cells

Cardiopulmonary and Laboratory Profiling of Patients with Thalassemia At Risk for Pulmonary Hypertension: Report From the Thalassemia Clinical Research Network.

Pan-Selectin Antagonist GMI-1070 Affects Biomarkers of Adhesion, Activation and the Coagulation Cascade in Sickle Cell Adults At Steady State

NO or No NO, Increased Reduction of Nitrite to Nitric Oxide by Modified Red Blood Cells

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American Society of Hematology

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