Blood

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

Blood

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

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.

Blood

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

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

Blood

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

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

Blood

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

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.

Blood

R. Clark Brown, Kimberly Cruz, Theodosia A. Kalfa, Frans A. Kuypers, Santosh L. Saraf, Jeremie H. Estepp, Luke R. Smart, Punam Malik, Mark Lerman, Renae Mayer, Maria D. Ribadeneira, Sanjeev Forsyth, Patricia Schroeder, Eric Wu, Patrick Kelly, Marilyn J. Telen

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.

Blood

Theodosia A. Kalfa, Frans A Kuypers, Marilyn J. Telen, Punam Malik, Diamantis G. Konstantinidis, Jeremy H. Estepp, Hyon J. Kim, Santosh L. Saraf, Lindsey Wilson, Maria D Ribadeneira, Sanjeev Forsyth, Patricia Schroeder, Adam Drake, Olga Polyanskaya, Patrick Kelly, Lukasz Biernat

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

Blood

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

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

Blood

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

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

Marina Cavazzana, Jean-Antoine Ribeil, Emmanuel Payen, Felipe Suarez, Yves Beuzard, Fabien Touzot, Resy Cavallesco, Francois Lefrere, Stany Chretien, Philippe Bourget, Fabrice Monpoux, Corinne Pondarre, Benedicte Neven, Manfred Schmidt, Christof von Kalle, Frans A. Kuypers, Laura Sandler, Sandeep Soni, Olivier Hermine, Stephane Blanche, Mariane De Montalembert, Salima Hacien-Bey-Abina, Philippe Leboulch

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