Introduction: Pyruvate kinase (PK) deficiency is a rare, congenital, hemolytic anemia caused by mutations in the PKLR gene. Patients, including those never managed with blood transfusions, experience iron overload and other disease-related complications. In 2024, the first international expert ...
The disclosure relates to modulating pyruvate kinase and provides novel chemical compounds of formula (I) useful as activators of PKR, as well as various uses of these compounds. PK
Alarm features such as weight loss, change in bowel habit, iron deficiency anaemia, rectal bleeding or a palpable abdominal mass have been proposed as the optimal method of identifying patients in primary care who need early assessment and investigations. However, the accuracy of these clinical ...