Results 11 to 20 of about 405 (131)

Luspatercept versus mitapivat for non‐transfusion‐dependent β‐thalassemia: Dare to compare? [PDF]

HemaSphere
HemaSphere, Volume 9, Issue 7, July 2025.
Khaled M. Musallam, Sujit Sheth, Maria Domenica Cappellini, Kevin H. M. Kuo, Antonis Kattamis, Yesim Aydinok, Vip Viprakasit, Ali T. Taher   +7 more
doaj   +5 more sources

Mitapivat for Acquired Pyruvate Kinase Deficiency [PDF]

Pediatric Blood and Cancer
ABSTRACTPyruvate kinase (PK) activation is emerging as a promising treatment modality for numerous congenital hemolytic anemias of diverse pathophysiology, and one agent, mitapivat, is already licensed to treat patients with congenital PK deficiency.
Hanny Al-Samkari
exaly   +4 more sources

Relative Bioavailability Studies With Mitapivat: Formulation and Food Effect Assessments in Healthy Subjects [PDF]

Clinical Pharmacology in Drug Development
AbstractPyruvate kinase (PK) deficiency is a rare, hereditary, hemolytic anemia caused by mutations in the PKLR gene encoding the PK enzyme. Mitapivat (previously designated AG‐348) is a first‐in‐class, oral, allosteric activator of PK. We report results from 5 Phase 1 trials in healthy adults to characterize and compare mitapivat pharmacokinetics ...
Iyer V, Sullivan K, Yan Y, Hawkins P.
exaly   +4 more sources

Mitapivat-Associated Adverse Effects and Potential Mechanistic: Insights From Real-World Data. [PDF]

J Cell Mol Med
Journal of Cellular and Molecular Medicine, Volume 29, Issue 19, October 2025.
Liang G, Liu Q.
europepmc   +5 more sources

Mitapivat: New dawn in pyruvate kinase deficiency and beyond

Pediatric Hematology Oncology Journal
Mitapivat is the first in class oral allosteric activator of pyruvate kinase enzyme, leading to increased ATP production. Since red blood cells (RBC) rely on anaerobic metabolism, converting phosphoenolpyruvate to pyruvate in Embden– Meyerhof glycolytic ...
Ritika Khurana, Sangeeta Mudaliar
doaj   +2 more sources

Potent Fluorescent Probe for Target-Engagement Studies of Allosteric Pyruvate Kinase Modulators. [PDF]

Angew Chem Int Ed Engl
LumiPK, a novel environment‐sensitive fluorescent tracer, enables direct monitoring of allosteric ligand engagement with pyruvate kinase fused with nanoluciferase (Nluc), focusing on the liver isoform (PKL). In NanoBRET, and fluorescence indicator displacement assays, LumiPK shows high affinity and validates known allosteric modulators of PKL.
Nilsson O, Valaka AP, Håversen L, Bogucka A, Köteles I, Brear P, Rutberg M, Gunnarsson A, Hyvönen M, Grøtli M.   +9 more
europepmc   +3 more sources

Mitapivat for phosphofructokinase deficiency. [PDF]

Am J Hematol
Al-Samkari H.
europepmc   +3 more sources

Post-translational Modifications in Proteins: Prediction Methods, Biological Functions, and Diseases. [PDF]

MedComm (2020)
Posttranslational modifications (PTMs) serve as rapid and reversible molecular switches that reshape protein activity, stability, and interactomes, thereby governing virtually all physiological cues ranging from signal transduction to epigenetic memory. Mass spectrometry‐based proteomics has substantially expanded our understanding of PTM emergence and
Zhang S, Li J, Chen M, Huang H.
europepmc   +2 more sources

S273: EVIDENCE OF NONINFERIORITY OF MITAPIVAT VERSUS SPLENECTOMY IN MURINE HEREDITARY SPHEROCYTOSIS

HemaSphere, 2022
A. Matte’, A. Recchiuti, E. Federti, P. A. Kosinski, V. Riccardi, I. Iatcenko, L. Dang, A. Iolascon, R. Russo, C. Brugnara, A. Janin, C. Leboeuf, N. Mohandas, L. De Franceschi   +13 more
doaj   +2 more sources

P1477: MITAPIVAT EFFICACY IN ADULTS WITH PYRUVATE KINASE DEFICIENCY AND BASELINE HEMOGLOBIN LEVELS >10 G/DL [PDF]

HemaSphere, 2023
Rachael F. Grace, Janet Kwiatkowski, Eduard van Beers, Feng Tai, Bryan Mcgee, Hanny Al-Samkari   +5 more
doaj   +2 more sources