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The Lancet. Haematology|February 13, 2025
Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent β-thalassaemia (BELIEVE): final results from a phase 3 randomised trialMaria Domenica Cappellini, Vip Viprakasit, Pencho Georgiev, et al.
The Lancet. Haematology|May 30, 2020
Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trialAurelio Maggio, Antonis Kattamis, Mariagrazia Felisi, et al.
The Lancet. Haematology|August 25, 2022
Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trialAli T Taher, Maria Domenica Cappellini, Antonis Kattamis, et al.
Blood|December 15, 2005
A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemiaMaria Domenica Cappellini, Alan Cohen, Antonio Piga, et al.
The New England Journal of Medicine|April 24, 2024
Exagamglogene Autotemcel for Transfusion-Dependent β-ThalassemiaFranco Locatelli, Peter Lang, Donna Wall, et al.
The Lancet. Haematology|July 14, 2023
Sickle cell disease landscape and challenges in the EU: the ERN-EuroBloodNet perspectiveMaría Del Mar Mañú Pereira, Raffaella Colombatti, Federico Alvarez, et al.
American Journal of Hematology|June 8, 2026
Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent β-ThalassemiaSujit Sheth, Selim Corbacioglu, Josu de la Fuente, et al.
The New England Journal of Medicine|December 7, 2020
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-ThalassemiaHaydar Frangoul, David Altshuler, M Domenica Cappellini, et al.
Blood|July 1, 2024
Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapyMaxwell E Chappell, Laura Breda, Lucas Tricoli, et al.
Hemasphere|November 3, 2025
Isatuximab in combination with chemotherapy for pediatric patients with relapsed/refractory acute lymphoblastic leukemia or acute myeloid leukemia: The ISAKIDS studyAndré Baruchel, Karsten Nysom, Hyoung Jin Kang, et al.
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