Results 71 to 80 of about 405 (131)

P1544: BONE MINERAL DENSITY REMAINS STABLE IN PYRUVATE KINASE DEFICIENCY PATIENTS RECEIVING LONG-TERM TREATMENT WITH MITAPIVAT

HemaSphere, 2022
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   +17 more
doaj   +1 more source

P1548: LONG-TERM TREATMENT WITH ORAL MITAPIVAT IS ASSOCIATED WITH NORMALIZATION OF HEMOGLOBIN LEVELS IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY

HemaSphere, 2022
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   +14 more
doaj   +1 more source

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

HemaSphere, 2022
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   +9 more
doaj   +1 more source

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 ALPHA- OR BETA-THALASSEMIA

HemaSphere, 2022
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   +13 more
doaj   +1 more source

P1735: IMPROVEMENTS IN PATIENT-REPORTED OUTCOMES IN MITAPIVAT-TREATED PATIENTS WITH PYRUVATE KINASE DEFICIENCY: A DESCRIPTIVE ANALYSIS FROM THE PHASE 3 ACTIVATE TRIAL

HemaSphere, 2022
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   +10 more
doaj   +1 more source

Pyruvate kinase activators for treatment of pyruvate kinase deficiency. [PDF]

Hematology Am Soc Hematol Educ Program, 2023
Grace RF.
europepmc   +1 more source

S116: LONG-TERM EFFICACY AND SAFETY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON—TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA

HemaSphere, 2022
K Kuo, D Layton, A Lal, H Al-Samkari, J Bhatia, P Kosinski, B Tong, M Lynch, K Uhlig, E Vichinsky   +9 more
doaj   +1 more source

Pyruvate kinase activators: targeting red cell metabolism in thalassemia. [PDF]

Hematology Am Soc Hematol Educ Program, 2023
Kuo KHM.
europepmc   +1 more source

One-year safety and efficacy of mitapivat in sickle cell disease: follow-up results of a phase 2, open-label study. [PDF]

Blood Adv, 2023
van Dijk MJ, Rab MAE, van Oirschot BA, Bos J, Derichs C, Rijneveld AW, Cnossen MH, Nur E, Biemond BJ, Bartels M, Jans JJM, van Solinge WW, Schutgens REG, van Wijk R, van Beers EJ.   +14 more
europepmc   +1 more source

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 NOT REGULARLY TRANSFUSED

HemaSphere, 2022
H. Al-Samkari, J. Morales-Arias, R. Xu, V. Beynon, R. F. Grace   +4 more
doaj   +1 more source