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Robert Liem
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Journal Articles

Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia

Free
Franco Locatelli, Roland Meisel, Selim Corbacioglu, Josu de la Fuente, Mattia Algeri, Joachim Rupprecht, Kevin Kuo, Ami Shah, Peter Lang, Hayley Merkeley, Ben Carpenter, Markus Mapara, Robert Liem, Stephan Grupp, Yogi Chopra, Amanda Li, Janet Kwiatkowski, Melanie Kirby-Allen, Maria Domenica Cappellini, Antonis Kattamis, Sujit Sheth, Kevin Boerner, Sakellarios Zairis, Tina Liu, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2025) 146 (Supplement 1): 6089.
https://doi.org/10.1182/blood-2025-6089
Published: 2025
Journal Articles

Long-term follow-up demonstrates durable clinical benefits of exagamglogene autotemcel for sickle cell disease with recurrent vaso-occlusive crises: Final results of climb SCD-121

Free
Stephan Grupp, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Mapara, Robert Liem, Laurence Dedeken, Lyndsay Molinari, Michael Eckrich, Kevin Kuo, Paul Telfer, Ami Shah, Marina Cavazzana, Selim Corbacioglu, Mariane de Montalembert, Stephan Lobitz, Damiano Rondelli, Martin Steinberg, Mark Walters, Kevin Boerner, Tina Liu, Suzan Imren, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2025) 146 (Supplement 1): 2537.
https://doi.org/10.1182/blood-2025-2537
Published: 2025
Journal Articles

Development of the sickle cell outcome grading system (SCOGS), a novel classification system for sickle cell disease severity

Free
Tarun Aurora, Renee Hodges, Joseph Wardell, Martha Barton, Janna Lipford, Clifford Takemoto, Marisol Betensky, Payal Desai, Seethal Jacob, Parul Rai, John Strouse, Lewis Hsu, Melissa Azul, Monica Hulbert, Winfred Wang, Ravi Talati, Robert Liem, Sheinei Alan, Elizabeth Klings, Abena Appiah-Kubi, Caterina Minniti, India Sisler, Patrick McGann, Madhav Vissa, Marquita Nelson, Stephanie Guarino, Robin Miller, Sharl Azar, Sherif Badawy, Christina Abrams, Sophie Lanzkron, Elna Saah, Farranz Alvarez Nunez, Nadirah El-Amin, Nickhill Bhakta, Jane Hankins
Journal: Blood
Blood (2025) 146 (Supplement 1): 2942.
https://doi.org/10.1182/blood-2025-2942
Published: 2025
Journal Articles

Return Visit Rates after an Emergency Department Treat-and-Release Visit for Children with Sickle Cell Pain Episodes

Free
Keli C Coleman, Kenneth McKinley, Angela Ellison, Amanda M. Brandow, Blake Nielsen, Lawrence Cook, Elizabeth Alpern, Selena Hariharan, Irina Topoz, Morgan Wurtz, Claudia R. Morris, Andrew D. Campbell, Robert Liem, Rachelle Nuss, Charles T. Quinn, Alexis A. Thompson, Anthony Villella, Ana Baumann, Allison A. King, Warren Frankenberger, David C Brousseau
Journal: Blood
Blood (2022) 140 (Supplement 1): 5418–5419.
https://doi.org/10.1182/blood-2022-166132
Published: 2022
Journal Articles

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Free
Franco Locatelli, Peter Lang, Amanda Li, Selim Corbacioglu, Josu de la Fuente, Donna A. Wall, Robert Liem, Roland Meisel, Markus Y. Mapara, Ami J. Shah, Maria Domenica Domenica Cappellini, Antonis Kattamis, Sujit Sheth, Yael Bobruff, Laura Bower, Lanju Zhang, Anjali Sharma, Yang Song, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2022) 140 (Supplement 1): 4899–4901.
https://doi.org/10.1182/blood-2022-166881
Published: 2022
Journal Articles

Prediction of High Acute Care Utilization in Children with Sickle Cell Disease

Free
Josiah Ballantine, Soyang Kwon, Robert Liem
Journal: Blood
Blood (2022) 140 (Supplement 1): 5129–5130.
https://doi.org/10.1182/blood-2022-168186
Published: 2022
Journal Articles

Efficacy and Safety of 1500 mg Voxelotor in a Phase 2a Study (GBT440-007) in Adolescents with Sickle Cell Disease

Free
Clark Brown, Carolyn Hoppe, Adlette Inati, Miguel R. Abboud, Winfred Wang, MD, Robert Liem, MD, Gerald Woods, Lewis L. Hsu, Victor R. Gordeuk, MD, Connie Piccone, Richard A. Drachtman, Erica Fong, Sandra Dixon, Margaret Tonda, PharmD, Carla Washington, Joshua Lehrer-Graiwer
Journal: Blood
Blood (2018) 132 (Supplement 1): 509.
https://doi.org/10.1182/blood-2018-99-117510
Published: 2018
Journal Articles

Initial Results from a Cohort in a Phase 2a Study (GBT440-007) Evaluating Adolescents with Sickle Cell Disease Treated with Multiple Doses of GBT440, a HbS Polymerization Inhibitor

Free
Carolyn C Hoppe, MD, Adlette C. Inati, MD, Clark Brown, MD PhD, Winfred Wang, MD, Victor R. Gordeuk, MD, Robert Liem, MD, Gerald Woods, MD, Connie M. Piccone, MD, Erica Fong, Ganesh Balaratnam, MD, Sandy Dixon, Margaret E Tonda, PharmD, Carla B Washington, PhD, Yifah Yaron, MD PhD, Josh Lehrer, MD MPhil, FACC
Journal: Blood
Blood (2017) 130 (Supplement 1): 689.
https://doi.org/10.1182/blood.V130.Suppl_1.689.689
Published: 2017
Journal Articles

Serum Ferritin and Liver Iron Burden by MRI Predict Cardiac Iron Overload in Thalassemia Major.

Free
Alexis A. Thompson, MD, R. Andrew De Freitas, MD, Robert Liem, MD, Dena Fasheh, MS, John C. Wood, MD, Cynthia K. Rigsby, MD
Journal: Blood
Blood (2006) 108 (11): 1771.
https://doi.org/10.1182/blood.V108.11.1771.1771
Published: 2006