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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

Janet L. Kwiatkowski, Mark C. Walters, Suradej Hongeng, Evangelia Yannaki, Andreas E. Kulozik, Joachim B. Kunz, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Tao Ge, Shamshad Ali, Himal L. Thakar, Heidi Elliot, Ankit Lodaya, Ji Hyun Lee, Richard A. Colvin, Franco Locatelli, Alexis A. Thompson

2024The Lancet38 citationsDOI

Topics & Concepts

Open labelMedicineInternal medicineRandomized controlled trialGenotypeThalassemiaBlood transfusionGeneBiologyGeneticsHemoglobinopathies and Related DisordersBlood groups and transfusionErythropoietin and Anemia Treatment
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 | Litcius