Ontology highlight
ABSTRACT:
SUBMITTER: Kung C
PROVIDER: S-EPMC5609468 | biostudies-literature | 2017 Sep
REPOSITORIES: biostudies-literature
Blood 20170731 11
Pyruvate kinase (PK) deficiency is a rare genetic disease that causes chronic hemolytic anemia. There are currently no targeted therapies for PK deficiency. Here, we describe the identification and characterization of AG-348, an allosteric activator of PK that 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 il ...[more]