Back to Search
Start Over
AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency.
- Source :
-
Blood . 9/14/2017, Vol. 130 Issue 11, p1347-1356. 10p. - Publication Year :
- 2017
-
Abstract
- Pyruvate kinase (PK) deficiency is a rare genetic disease that causes chronic hemolytic anemia. There are currently no targeted therapies for PKdeficiency. Here,we describe the identification and characterization of AG-348, an allosteric activator ofPKthat is currently in clinical trials for the treatment of PK deficiency. We demonstrate that AG-348 can increase the activity of wild-type and mutant PK enzymes in biochemical assays and in patient red blood cells treated ex vivo. These data illustrate the potential for AG-348 to restore the glycolytic pathway activity in patients withPKdeficiencyand ultimately lead to clinical benefit. [ABSTRACT FROM AUTHOR]
- Subjects :
- *HEMOLYTIC anemia
*PYRUVATE kinase
*ERYTHROCYTES
*GLYCOLYSIS
*CLINICAL trials
Subjects
Details
- Language :
- English
- ISSN :
- 00064971
- Volume :
- 130
- Issue :
- 11
- Database :
- Academic Search Index
- Journal :
- Blood
- Publication Type :
- Academic Journal
- Accession number :
- 125165968
- Full Text :
- https://doi.org/10.1182/blood-2016-11-753525