Companies in #Rare Hematology
Found 1 company operating in this sector.
Agios Pharmaceuticals is a biopharmaceutical company focused on developing therapies for rare diseases, including pyruvate kinase (PK) activation and other cellular metabolism targets. The company markets PYRUKYND® (mitapivat) for hemolytic anemia in adults with PK deficiency and is exploring additional indications in thalassemia and sickle cell disease.