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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

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

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

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

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

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

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

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

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

A Family with Hereditary Elliptocytosis: Variable Clinical Severity Caused by Three Mutations in the α-Spectrin Gene,

Paul Hubert Frans Franck, PhD, Cobie Postma, Marjan Veuger, PhD, Pierre Wijermans, MD, Frans A Kuypers, PhD

Blood (2011) 118 (21): 3167.

https://doi.org/10.1182/blood.V118.21.3167.3167

Published: 2011

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

Journal Articles

Effect of Short-Term Simvastatin Treatment on Endothelial Adhesion Molecules in Sickle Cell Disease: A Pilot Study

Carolyn Hoppe, MD, Frans A Kuypers, PhD, Sandra K Larkin, Robert Hagar, MD, Elliott Vichinsky, MD, Lori A Styles, MD

Blood (2011) 118 (21): 1070.

https://doi.org/10.1182/blood.V118.21.1070.1070

Published: 2011

Journal Articles

A Pilot Study of Tapered Oral Dexamethasone for the Acute Chest Syndrome of Sickle Cell Disease.

Charles T. Quinn, MD, MS, Marie J. Stuart, MD, Karen Kesler, PhD, Kenneth I. Ataga, MD, Winfred C. Wang, MD, Lori Styles, MD, Kim Smith-Whitley, MD, Ted Wun, MD, Suba Krishnan, MD, Frans A. Kuypers, PhD, Yamaja Setty, PhD, Nigel S. Key, MB, ChB, Seungshin Rhee, MS, George R. Buchanan, MD

Blood (2009) 114 (22): 1515.

https://doi.org/10.1182/blood.V114.22.1515.1515

Published: 2009

Journal Articles

Improved Engraftment without Graft-Versus-Host Disease After MHC-Mismatched Cord Blood Transplantation with Photochemically Treated Donor Lymphocytes.

Bindu Kanathezhath, MD, Myra Mizokami, MD, PhD, Lynne Neumayr, M.D., Hua Guo, MD, Mark C. Walters, MD, Frans A. Kuypers, PhD

Blood (2009) 114 (22): 2425.

https://doi.org/10.1182/blood.V114.22.2425.2425

Published: 2009

NARROW

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

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

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

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

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

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

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.

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

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

A Family with Hereditary Elliptocytosis: Variable Clinical Severity Caused by Three Mutations in the α-Spectrin Gene,

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

Effect of Short-Term Simvastatin Treatment on Endothelial Adhesion Molecules in Sickle Cell Disease: A Pilot Study

A Pilot Study of Tapered Oral Dexamethasone for the Acute Chest Syndrome of Sickle Cell Disease.

Improved Engraftment without Graft-Versus-Host Disease After MHC-Mismatched Cord Blood Transplantation with Photochemically Treated Donor Lymphocytes.

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