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P-055: TRIAL IN PROGRESS: A PHASE 2, OPEN-LABEL STUDY EVALUATING THE SAFETY AND EFFICACY OF THE ERYTHROCYTE PYRUVATE KINASE ACTIVATOR ETAVOPIVAT IN PATIENTS WITH THALASSEMIA OR SICKLE CELL DISEASE.

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COATES T., LAL A., BROWN R., KALFA T., KWIATKOWSKI J., BREVARD J., TRENOR C., WOOD K., SHETH S.

S270: LONGER-TERM ANALYSIS OF EFFICACY OF LUSPATERCEPT VERSUS PLACEBO IN PATIENTS WITH TRANSFUSION-DEPENDENT BETA-THALASSEMIA ENROLLED IN THE BELIEVE STUDY.

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M. D. Cappellini, A. T. Taher, J. B. Porter, K. H. Kuo, T. D. Coates, E. Voskaridou, G. L. Forni, S. Perrotta, A. Khelif, A. Lal, A. Kattamis, A. Piga, O. Hermine, N. Holot, F. Lersch, J. K. Shetty, S. Vodala, J. Zhang, D. Miteva, V. Viprakasit

PB2232: TRIAL IN PROGRESS: A PHASE 2, OPEN-LABEL STUDY EVALUATING THE SAFETY AND EFFICACY OF THE ERYTHROCYTE PYRUVATE KINASE ACTIVATOR ETAVOPIVAT IN PATIENTS WITH THALASSEMIA OR SICKLE CELL DISEASE.

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A. Lal, R. C. Brown, T. Coates, T. Kalfa, J. Kwiatkowski, J. Brevard, C. Trenor, K. Wood, S. Sheth

P1522: 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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K. H. M. Kuo, D. M. Layton, A. Lal, H. Al-Samkari, J. Bhatia, P. A. Kosinski, B. Tong, M. Lynch, K. Uhlig, E. P. Vichinsky

P1518: LONG-TERM SAFETY RESULTS OF THE BELIEVE STUDY OF LUSPATERCEPT IN ADULTS WITH ?ETA-THALASSEMIA.

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V. Viprakasit, M. D. Cappellini, J. B. Porter, K. H. M. Kuo, T. D. Coates, E. Voskaridou, V. M. Pinto, I. Tartaglione, A. Khelif, A. Lal, A. Kattamis, A. Piga, O. Hermine, N. Holot, F. Lersch, J. K. Shetty, J. Zhang, L. Bueno, A. C. Giuseppi, D. Miteva, A. T. Taher

S116: 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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K Kuo, D Layton, A Lal, H Al-Samkari, J Bhatia, P Kosinski, B Tong, M Lynch, K Uhlig, E Vichinsky

S103: 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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A. Lal, C. Brown, T. Coates, T. Kalfa, J.L. Kwiatkowski, J. Brevard, C. Trenor, K. Wood, S. Sheth

RESULTS FROM THE PHASE 3 NORTHSTAR-3 STUDY EVALUATING LENTIGLOBIN GENE THERAPY IN PATIENTS WITH TRANSFUSION-DEPENDENT ß-THALASSAEMIA AND A ß0 OR IVS-I-110 MUTATION AT BOTH ALLELES OF THE HBB GENE.

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A. E. Kulozik, F. Locatelli, E. Yannaki, J. B. Porter, I. Thuret, M. G. Sauer, A. Lal, J. L. Kwiatkowski, H. Elliot, G. Tao, R. A. Colvin, A. A. Thompson

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