Open accessReview articleFirst published 2026-01-01Translational insights from nonclinical studies of AAV gene therapies for hemophilia: mechanisms underpinning variability and durability of gene expression Sylvia FongORCID, Laura L. SwystunORCID, Paul BattyORCID , [...]View all
Open accessResearch articleFirst published 2026-01-01Endothelial dysfunction and cardiac damage indicators in patients with β-thalassemia major under iron-chelation therapy Ola M. Al-SanabraORCID, Manal A. AbbasORCID, Wafà J. Hazà , [...]View all
Open accessResearch articleFirst published 2026-01-01Efficacy and safety of chimeric antigen receptor T-cell therapy in relapsed/refractory large B-cell lymphoma: a systematic review and meta-analysis Qiang Li, Shenghua Xu, Yun Zhong , [...]View all
Open accessResearch articleFirst published 2026-01-01Outcomes in participants switching from FVIII replacement therapy to efanesoctocog alfa prophylaxis in XTEND-1: a post hoc analysis Sophie SusenORCID, Roshni Kulkarni, Keiji Nogami , [...]View all