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Gene therapies for sickle cell disease: final evidence report
Gene therapies for sickle cell disease: final evidence report
We reviewed the clinical effectiveness of lovotibeglogene autotemcel (lovo-cel) and exagamglogene autotemcel (exa-cel) versus standard of care for the treatment of severe sickle cell disease. We also intended to compare lovo-cel to exa-cel via any evidence directly comparing the two therapies or by indirect comparisons of key outcomes. We sought evidence on patient-important outcomes, including resolution of vaso-occlusive crisis, health-related quality of life, and adverse events.
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