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...

Full description

Bibliographic Details

Main Authors:	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
Format:	Artigo
Language:	Inglês
Published:	Elsevier 2025-12-01
Series:	Blood Advances
Online Access:	http://www.sciencedirect.com/science/article/pii/S2473952925005038
Tags:	Add Tag No Tags, Be the first to tag this record!