Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial - Kwiatkowski, Janet; Walters, Mark C; Hongeng, Suradej; Yannaki, Evangelia; Kulozik, Andreas; Kunz, Joachim; Sauer, Martin G; Thrasher, Adrian J; Thuret, Isabelle; Lal, Ashutosh; Tao, Ge; Ali, Shamshad; et al. (The lancet, 2024)
Online verfügbar: 8. November 2024, Artikelversion: 29. November 2024