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Journal Articles

Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia

Alexis Thompson, Janet L. Kwiatkowski, Jennifer Schneiderman, Isabelle Thuret, Andreas E. Kulozik, Evangelia Yannaki, Marina Cavazzana, Suradej Hongeng, Timothy S. Olson, Martin G. Sauer, Adrian J. Thrasher, Ashutosh Lal, John E. J. Rasko, Shamshad Ali, Ge Tao, Himal L. Thakar, Ami Deora, Clark Paramore, Niki Witthuhn, Mark C. Walters, Franco Locatelli
Journal: Blood
Blood (2024) 144 (Supplement 1): 2194.
https://doi.org/10.1182/blood-2024-200152
Published: 2024
Journal Articles

Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up

Janet L. Kwiatkowski, Timothy S. Olson, Mark C. Walters, John B. Porter, Jennifer Schneiderman, Suradej Hongeng, Andreas Kulozik, Marina Cavazzana, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, John E. J. Rasko, Evangelia Yannaki, Shamshad Ali, Gloria Tao, Ami Deora, Himal L. Thakar, Richard A. Colvin, Franco Locatelli, Alexis A. Thompson
Journal: Blood
Blood (2023) 142 (Supplement 1): 2480.
https://doi.org/10.1182/blood-2023-173869
Published: 2023
Journal Articles

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Alexis A. Thompson, Timothy S. Olson, Mark C. Walters, John B. Porter, Jennifer Schneiderman, Suradej Hongeng, Andreas Kulozik, Marina Cavazzana, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, John E. J. Rasko, Evangelia Yannaki, Shamshad Ali, Gloria Tao, Himal L. Thakar, Ami Deora, Katiana Gruppioni, Richard A. Colvin, Franco Locatelli, Janet L. Kwiatkowski
Journal: Blood
Blood (2023) 142 (Supplement 1): 1102.
https://doi.org/10.1182/blood-2023-174068
Published: 2023
Journal Articles

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Mark C. Walters, Janet L. Kwiatkowski, John B. Porter, Jennifer Schneiderman, Suradej Hongeng, Andreas E. Kulozik, Marina Cavazzana, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, John E.J. Rasko, Evangelia Yannaki, Shamshad Ali, Ilya Shestopalov, Maeva Fincker, Richard A. Colvin, Dustin Whitney, Franco Locatelli, Alexis A. Thompson
Journal: Blood
Blood (2022) 140 (Supplement 1): 5355–5357.
https://doi.org/10.1182/blood-2022-162400
Published: 2022
Journal Articles

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Franco Locatelli, Mark C. Walters, Janet L. Kwiatkowski, Marina Cavazzana, Suradej Hongeng, John B. Porter, Adrian J. Thrasher, Andreas E. Kulozik, Isabelle Thuret, John E.J. Rasko, Evangelia Yannaki, Martin G. Sauer, Shamshad Ali, Himal Thakar, Katiana Gruppioni, Alexis A. Thompson
Journal: Blood
Blood (2022) 140 (Supplement 1): 8200–8202.
https://doi.org/10.1182/blood-2022-163178
Published: 2022