Kwiatkowski JL, Walters MC, Hongeng S, Yannaki E, et al. Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype beta-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. Lancet 2024;404:2175-2186.
PMID: 39527960