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

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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

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

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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

Pharmacokinetics (PK) of Deferasirox in Transfusion Dependent Thalassemia.

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Beth Apsel Winger, Felicity Usac, Liusheng Huang, Ashutosh Lal, Janel R. Long-Boyle

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

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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

Mitapivat Improves Markers of Erythropoietic Activity in Long-Term Study of Adults with Alpha- or Beta-Non-Transfusion-Dependent Thalassemia.

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Kevin H.M. Kuo, D. Mark Layton, Ashutosh Lal, Hanny Al-Samkari, Penelope A. Kosinski, Bo Tong, Jeremie H. Estepp, Katrin Uhlig, Elliott P. Vichinsky

Long Term Outcomes of 63 Patients with Transfusion-Dependent ß-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

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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

Trial in Progress: A Phase 2, Open-Label Study Evaluating the Safety and Efficacy of the PKR Activator Etavopivat (FT-4202) in Patients with Thalassemia or Sickle Cell Disease.

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Ashutosh Lal, R. Clark Clark Brown, Thomas D. Coates, Theodosia A. Kalfa, Janet L. Kwiatkowski, Julie Brevard, Von Potter, Ken W. Wood, Sujit Sheth

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up.

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Alexis A. Thompson, Franco Locatelli, Evangelia Yannaki, Mark C. Walters, John B. Porter, Suradej Hongeng, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Marina Cavazzana, John E.J. Rasko, Lin Du, Richard A. Colvin, Janet L. Kwiatkowski

Long-Term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia.

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Kevin H.M. Kuo, D. Mark Layton, Ashutosh Lal, Hanny Al-Samkari, Joy Bhatia, Penelope A. Kosinski, Bo Tong, Megan Lynch, Katrin Uhlig, Elliott Vichinsky

Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent ß-Thalassemia Enrolled in Phase 3 Studies.

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Janet L. Kwiatkowski, Franco Locatelli, Mark C. Walters, John B. Porter, Suradej Hongeng, Evangelia Yannaki, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Ruiting Guo, Richard A. Colvin, Katiana Gruppioni, Alexis A. Thompson

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