Results 21 to 30 of about 405 (131)

P1497: MITAPIVAT IMPROVES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED [PDF]

HemaSphere, 2023
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   +11 more
doaj   +2 more sources

Long‐term efficacy and safety of mitapivat in non‐transfusion‐dependent α‐ or β‐thalassaemia: An open‐label phase 2 study [PDF]

British Journal of Haematology
Hanny Al-Samkari, Elliott Vichinsky, Kevin H M Kuo   +2 more
exaly   +2 more sources

Advances in Human Mass Balance Studies: An IQ Consortium Perspective on Current Practices and Emerging Trends. [PDF]

Clin Pharmacol Ther
Human radiolabeled mass balance studies are crucial for comprehensively characterizing the absorption, distribution, metabolism, and excretion (ADME) of investigational drugs, providing essential data for drug development, regulatory evaluation, and product labeling.
Boer J, Cannady E, Cuyckens F, Glaenzel U, Granvil C, Huth F, James AD, Li W, Veau C, Young G, Yue Q, Walker GS, Argikar UA, Bauman JN, Cantalloube C, Chen YL, Kang P, Moliner P, Pedersen ML, Piel I, Schadt S, Schieferstein H, Suchomel J, Wang S, Khojasteh SC.   +24 more
europepmc   +2 more sources

RESULTS FROM A PHASE 2 STUDY OF MITAPIVAT IN ADULTS WITH NON–TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA

Hematology, Transfusion and Cell Therapy, 2021
Objectives: Thalassemias are characterized by ineffective erythropoiesis and hemolysis due to imbalanced production and precipitation of globin chains.
KH Kuo, DM Layton, A Lal, H Al-Samkari, J Bhatia, B Tong, M Lynch, K Uhlig, EP Vichinsky   +8 more
doaj   +1 more source

Mitapivat for sickle cell disease: the time is ripe for a new therapy. [PDF]

Ann Transl Med
Cheng AN, Al-Samkari H.
europepmc   +2 more sources

Mitapivat therapy in a patient with nonsense homozygous mutation of the Pyruvate Kinase L/R gene: a case report. [PDF]

Blood Transfus
Pyruvate kinase deficiency causes chronic hereditary non-spherocytic hemolytic anemia and is associated with long-term complications including iron overload, liver cirrhosis, endocrinopathies, osteoporosis, and pulmonary hypertension. Mitapivat is a first-in-class oral allosteric activator of erythrocyte-specific isoforms of the PK enzyme shown to ...
Ceglie S, Valentini CG, Pellegrino C, Massini G, Teofili L.   +4 more
europepmc   +2 more sources

Erythropoiesis in health and disease: Distinguishing defective and ineffective erythropoiesis. [PDF]

Hemasphere
Abstract Erythropoiesis is a finely regulated process ensuring continuous red blood cell production to maintain oxygen delivery. Disruptions in this process give rise to defective erythropoiesis, characterized by impaired lineage commitment and progenitor development, and ineffective erythropoiesis (IE), marked by expansion of erythroid progenitors ...
El Hoss S, Lizarralde-Iragorri MA, Maciel TT, Hermine O.   +3 more
europepmc   +2 more sources

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

Hematology, Transfusion and Cell Therapy, 2021
Objectives: Thalassemias are characterized by imbalanced globin-chain production resulting in excess α- or β-globin precipitation, hemolytic anemia, and ineffective erythropoiesis.
KH Kuo, DM Layton, H Al-Samkari, A Kattamis, S Sheth, A Taher, V Viprakasit, CX Chamberlain, L Czapla, S Gheuens, L Jiang, M Lynch, B Tong, K Uhlig, MD Cappellini   +14 more
doaj   +1 more source

P1458: MITAPIVAT AMELIORATES IN VITRO HUMAN Β THALASSEMIC ERYTHROID MATURATION INDEX AND MODULATES THE EXPRESSION OF THE CYTOPROTECTOR PEROXIREDOXIN-2. [PDF]

HemaSphere, 2023
Alessandro Matte’, Richard Pozzetto Huot, Andrea Mattarei, Alberto Ongaro, Alice Passarini, Angela Siciliano, Roberta Russo, Achille Iolascon, Carlo Brugnara, Lucia De Franceschi   +9 more
doaj   +2 more sources

Mitapivat: An Overview

, 2023
AbstractMitapivat is used to treat hemolytic anemia (a condition in which more red blood cells aredestroyed than made into the body) in people with pyruvate kinase deficiency. Mitapivat is ina class of drugs called pyruvate kinase activators .mitapivat increases pyruvate kinase whichresults in increment in lifespan of red blood cells.
Vani Madaan, Vandana Sharma, Ishita Gupta, Janvi Deshmukh, Rahul Gupta   +4 more
openaire   +1 more source