P1485: PKM AND PKR EXPRESSION DURING HEMATOPOIESIS AND ERYTHROPOIESIS by Erin Tsai, Leonardo Rivadeneyra, Saliha Yilmaz, Lenny Dang, Megan Wind-Rotolo

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PB2247: PHARMACOKINETIC MODELING AND SIMULATION TO SUPPORT MITAPIVAT DOSE SELECTION USED IN PEDIATRIC PHASE III STUDIES by O. Yin, K. Baron, J. Mondick, M. Little, P. D. Tyler, V. Beynon

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P1546: ACTIVATE-KIDST: MITAPIVAT IN CHILDREN WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED by R. F. Grace, P. D. Tyler, M. Little, P. A. Kosinski, V. Beynon

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P112: ENERGIZE AND ENERGIZE-T: TWO PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDIES OF MITAPIVAT IN ADULTS WITH NON--TRANSFUSION-DEPENDENT OR TRANSFUSION-DEPENDENT ALP... by K Kuo, D Layton, H Al-Samkari, A Kattamis, S Sheth, A Taher, V Viprakasit, C Chamberlain, L Czapla, S Gheuens, M Lynch, B Tong, K Uhlig, M Cappellini

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P1547: ACTIVATE-KIDS: MITAPIVAT IN CHILDREN WITH PYRUVATE KINASE DEFICIENCY WHO ARE NOT REGULARLY TRANSFUSED by R. F. Grace, P. D. Tyler, M. Little, P. A. Kosinski, V. Beynon

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S116: LONG-TERM EFFICACY AND SAFETY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON—TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA by K Kuo, D Layton, A Lal, H Al-Samkari, J Bhatia, P Kosinski, B Tong, M Lynch, K Uhlig, E Vichinsky

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P1522: LONG-TERM EFFICACY AND SAFETY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON–TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA by 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

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P1548: LONG-TERM TREATMENT WITH ORAL MITAPIVAT IS ASSOCIATED WITH NORMALIZATION OF HEMOGLOBIN LEVELS IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY by W. Barcellini, R. F. Grace, H. Al-Samkari, A. Glenthøj, J. A. Rothman, M. Morado Arias, D. M. Layton, O. Andres, M. DiBacco, P. Hawkins, M. P. Judge, F. Tai, J. Morales-Arias, V. Beynon, E. J. van Beers

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PB2547: ESTIMATING HEALTH-RELATED QUALITY OF LIFE IN ADULT PYRUVATE KINASE DEFICIENCY: A TIME TRADE-OFF UTILITY STUDY by Sara Higa, Gin Nie Chua, Anna-Katrine Sussex, Andrew Lloyd, Junlong LI, Parija Patel, Susan Morris, Erin Zagadailov

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P1497: MITAPIVAT IMPROVES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED by Eduard van Beers, Hanny Al-Samkari, Rachael F. Grace, Wilma Barcellini, Andreas Glenthøj, Vanessa Beynon, Megan Wind-Rotolo, Rengyi Xu, Melissa Dibacco, Parija Patel, John B. Porter, Kevin H.M. Kuo

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P1565: MITAPIVAT IMPROVES INEFFECTIVE ERYTHROPOIESIS AND REDUCES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY by E. J. van Beers, H. Al-Samkari, R. F. Grace, W. Barcellini, A. Glenthøj, M. P. Judge, P. A. Kosinski, R. Xu, V. Beynon, B. McGee, J. B. Porter, K. H. M. Kuo

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P1544: BONE MINERAL DENSITY REMAINS STABLE IN PYRUVATE KINASE DEFICIENCY PATIENTS RECEIVING LONG-TERM TREATMENT WITH MITAPIVAT by H. Al-Samkari, R. F. Grace, A. Glenthøj, O. Andres, W. Barcellini, F. Galactéros, K. H. M. Kuo, D. M. Layton, M. Morado Arias, V. Viprakasit, Y. Dong, F. Tai, L. Grekas, K. Khoja, S. Gheuens, B. McGee, J. B. Porter, E. J. van Beers

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P1545: DURABILITY OF HEMOGLOBIN RESPONSE AND REDUCTION IN TRANSFUSION BURDEN IS MAINTAINED OVER TIME IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT IN A LONG-TE... by R. F. Grace, A. Glenthøj, W. Barcellini, M. Verhovsek, J. A. Rothman, M. Morado Arias, D. M. Layton, O. Andres, F. Galactéros, E. J. van Beers, K. Onodera, V. Viprakasit, S. Chonat, J. B. Porter, M. P. Judge, P. A. Kosinski, P. Hawkins, S. Gheuens, R. Xu, B. McGee, V. Beynon, H. Al-Samkari

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P107: A PHASE 2/3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF MITAPIVAT IN PATIENTS WITH SICKLE CELL DISEASE by J Howard, K Kuo, A Oluyadi, H Shao, S Morris, A Zaidi, E Van Beers, S Thein

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P1543: MITAPIVAT DECREASES THE NEED FOR TRANSFUSIONS SECONDARY TO POORLY TOLERATED ANEMIA AND ACUTE EVENTS COMPARED TO PLACEBO IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE N... by H. Al-Samkari, J. Morales-Arias, R. Xu, V. Beynon, R. F. Grace

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P1735: IMPROVEMENTS IN PATIENT-REPORTED OUTCOMES IN MITAPIVAT-TREATED PATIENTS WITH PYRUVATE KINASE DEFICIENCY: A DESCRIPTIVE ANALYSIS FROM THE PHASE 3 ACTIVATE TRIAL by K. H. Kuo, R. F. Grace, E. J. van Beers, W. Barcellini, A. Glenthøj, S. Holzhauer, P. Patel, V. Beynon, J. Li, E. Zagadailov, H. Al-Samkari

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PI-05: PHARMACODYNAMIC EFFECTS OF AG-946, A HIGHLY POTENT NOVEL ACTIVATOR OF PYRUVATE KINASE, IN EX VIVO TREATMENT OF RED BLOOD CELLS FROM SICKLE CELL DISEASE PATIENTS by RAB M., VAN DIJK M., BOS J., VAN OIRSCHOT B., GERRITS J., KOSINSKI P., KUNG C., JANS J., VAN BEERS E., DANG L., VAN WIJK R.

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P1492: PHARMACODYNAMIC EFFECTS OF AG-946, A HIGHLY POTENT NOVEL ACTIVATOR OF PYRUVATE KINASE, IN EX VIVO TREATMENT OF RED BLOOD CELLS FROM SICKLE CELL DISEASE PATIENTS by M. Rab, M. van Dijk, J. Bos, B. van Oirschot, J. Gerrits, P. Kosinski, C. Kung, J. Jans, E. van Beers, L. Dang, R. van Wijk

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P1473: CLINICALLY RELEVANT HEMOGLOBIN RESPONSE IN ADULTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT – A SUB-ANALYSIS OF THE ACTIVATE TRIAL by Hanny Al-Samkari, Rachael F. Grace, Andreas Glenthøj, Wilma Barcellini, Madeleine Verhovsek, Jennifer A. Rothman, Marta Morado, Mark Layton, Oliver Andres, Frédéric Galactéros, Koichi Onodera, Satheesh Chonat, Rengyi Xu, Bryan Mcgee, Melissa Dibacco, Jaime Morales, Eduard van Beers

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MAT2A inhibitor AG-270/S095033 in patients with advanced malignancies: a phase I trial by Mrinal Gounder, Melissa Johnson, Rebecca S. Heist, Geoffrey I. Shapiro, Sophie Postel-Vinay, Frederick H. Wilson, Elena Garralda, Gerburg Wulf, Caroline Almon, Salah Nabhan, Elia Aguado-Fraile, Peng He, Mathilde Romagnoli, Mohammad Hossain, Rohini Narayanaswamy, Amel Sadou-Dubourgnoux, Michael Cooper, Vasileios Askoxylakis, Howard A. Burris, Josep Tabernero

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