Results 101 to 110 of about 405 (131)

Mitapivat versus Placebo for Pyruvate Kinase Deficiency

New England Journal of Medicine, 2022
Pyruvate kinase deficiency is a rare, hereditary, chronic condition that is associated with hemolytic anemia. In a phase 2 study, mitapivat, an oral, first-in-class activator of erythrocyte pyruvate kinase, increased the hemoglobin level in patients with pyruvate kinase deficiency.In this global, phase 3, randomized, placebo-controlled trial, we ...
Hanny Al-Samkari, Rachael Grace, Eduard Van Beers   +2 more
exaly   +12 more sources

Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

New England Journal of Medicine, 2019
Pyruvate kinase deficiency is caused by mutations in PKLR and leads to congenital hemolytic anemia. Mitapivat is an oral, small-molecule allosteric activator of pyruvate kinase in red cells.In this uncontrolled, phase 2 study, we evaluated the safety and efficacy of mitapivat in 52 adults with pyruvate kinase deficiency who were not receiving red-cell ...
Janet Kwiatkowski, Rachael Grace, Eduard Van Beers   +2 more
exaly   +5 more sources

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Mitapivat for sickle cell disease and thalassemia

Drugs of Today, 2023
Mitapivat, an oral first-in-class activator of erythrocyte pyruvate kinase (PKR), was first investigated in patients with pyruvate kinase deficiency (PKD), where it was found to improve hemoglobin (Hb) concentrations in patients who did not regularly receive transfusions and to reduce transfusion burden in patients who receive regular transfusions.
Federica, Pilo, Emanuele, Angelucci
openaire   +2 more sources

An evaluation of mitapivat for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency

Expert Review of Hematology, 2022
Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemia. Until recently, treatment had been limited to supportive management including red blood cell transfusions, splenectomy, and management of chronic disease complications such as iron overload and decreased bone mineral density.We discuss preclinical ...
Hanny Al-Samkari, Andrew Song
exaly   +3 more sources

Mitapivat: A Review in Pyruvate Kinase Deficiency in Adults

Drugs, 2023
Mitapivat (Pyrukynd®), an oral, allosteric activator of pyruvate kinase (PK), is approved in the USA for the treatment of haemolytic anaemia in adults with PK deficiency and in the EU and UK for the treatment of PK deficiency in adults. Mitapivat acts by restoring activity of the red blood cell (RBC) PK enzyme, which is dysfunctional due to genetic ...
Amy, Zhuang-Yan, Matt, Shirley
openaire   +2 more sources

Mitapivat for non-transfusion-dependent thalassaemia

Lancet, The
Selim Corbacioglu
exaly   +3 more sources

Efficacy and safety of Mitapivat in sickle cell disease: a systematic review

Expert Opinion on Pharmacotherapy
Sickle cell disease (SCD) is a chronic hemoglobinopathy marked by hemolytic anemia, VOEs, and multiorgan complications. Mitapivat, an oral Pyruvate Kinase (PK) activator, has emerged as a potential disease-modifying therapy. This systematic review assessed the efficacy and safety of Mitapivat in individuals with SCD.
Nazli Khatib, Deepak Saxena
exaly   +3 more sources

„First-in-Class“: Mitapivat hilft bei Pyruvatkinasemangel

Transfusionsmedizin, 2022
Der oral verfügbare Aktivator der Pyruvatkinase Mitapivat ist der erste als Arzneimittel verfügbare Vertreter dieser Wirkstoffklasse. In der doppelblinden, placebokontrollierten Studie überprüften Al-Samkari et al. die Wirksamkeit und Sicherheit bei Patienten, die nicht
openaire   +1 more source

Synthetic development of Mitapivat via improved route

Journal of Molecular Structure
Faraz Ghous, Ranjitsinh C Dabhi, Jayesh Maru   +2 more
exaly   +2 more sources