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Journal Articles

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

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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

First results of exagamglogene autotemcel in pediatric patients aged 5-11 years with transfusion-dependent β-thalassemia or sickle cell disease with recurrent severe vaso-occlusive crises

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Haydar Frangoul, Josu de la Fuente, Mattia Algeri, Yogi Chopra, Persis Amrolia, Akshay Sharma, Roland Meisel, Maria Domenica Cappellini, Selim Corbacioglu, Antonis Kattamis, Stephan Lobitz, Mariane de Montalembert, Damiano Rondelli, Sujit Sheth, Martin Steinberg, Mark Walters, Kevin Boerner, Katie Ender, Tina Liu, William Hobbs, Stephan Grupp, Franco Locatelli
Journal: Blood
Blood (2025) 146 (Supplement 1): 379.
https://doi.org/10.1182/blood-2025-379
Published: 2025