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Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel

26 Aug 2025

Abstract excerpt

ABSTRACT: Severe sickle cell disease (SCD), an inherited blood disorder characterized by recurrent vaso-occlusive crises (VOCs), significantly impairs health-related quality of life (HRQoL). Exagamglogene autotemcel (exa-cel), a one-time, ex vivo CRISPR/Cas9 gene-edited cell therapy, eliminated VOCs in 97% of participants with severe SCD in the phase 3 CLIMB SCD-121 trial. Here, we describe changes in...

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PMID: 40857358
DOI: 10.1182/bloodadvances.2025016701

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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