Concepedia

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,

The Lancet · 2024 · 38 citations · 14 references

DOI

Concepts

References

14

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

Alexis A. Thompson, Mark C. Walters, Janet L. Kwiatkowski et al. · New England Journal of Medicine · 2018 · 661 citations · Full text

, , +16

Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT)

Maria Domenica Cappellini, Alan R. Cohen, John B. Porter et al. · 2014 · 403 citations

, , +10

Betibeglogene Autotemcel Gene Therapy for Non–β <sup>0</sup> /β <sup>0</sup> Genotype β-Thalassemia

Franco Locatelli, Alexis A. Thompson, Janet L. Kwiatkowski et al. · New England Journal of Medicine · 2021 · 258 citations · Full text

, , +17

Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000–2010

D Baronciani, Emanuele Angelucci, Ulrike Pötschger et al. · Bone Marrow Transplantation · 2016 · 230 citations

, , +11

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Franco Locatelli, Peter Lang, Donna A. Wall et al. · New England Journal of Medicine · 2024 · 186 citations

, , +17