Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel

Abstract: Transfusion-dependent β-thalassemia (TDT) can have negative impacts on a patient’s health-related quality of life (HRQoL). Exagamglogene autotemcel (exa-cel) is a one-time, ex vivo CRISPR-Cas9 gene–edited cell therapy for TDT shown in a phase 3 clinical trial to result in transfusion indep...

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Autores principales:	Josu de la Fuente, Haydar Frangoul, Peter Lang, Donna Wall, Roland Meisel, Selim Corbacioglu, Amanda M. Li, Ami J. Shah, Ben Carpenter, Janet L. Kwiatkowski, Markus Y. Mapara, Robert I. Liem, Joachim Rupprecht, Kevin H.M. Kuo, Hayley Merkeley, Mattia Algeri, Wally Smith, Puja Kohli, Nanxin Li, Jaime Rubin, Siyu Zhang, William Hobbs, Franco Locatelli
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	Elsevier 2025-12-01
Colección:	Blood Advances
Acceso en línea:	http://www.sciencedirect.com/science/article/pii/S2473952925005038
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