THE hemolytic anemia associated with erythrocyte pyruvate kinase deficiency is of variable severity 1–17 and probably of variable cause. In some cases severe aberrations of erythrocyte shape ...

The following is a summary of “Pyruvate Kinase Function Correlates With Red Blood Cell Properties and Clinical Manifestations ...

Acute Hemolysis: Acute hemolysis with subsequent anemia has been observed ... Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease ...

The inducement grants consisted of (i) a nonstatutory option to purchase up to 55,374 shares of common stock, (ii) restricted stock units for 24,300 shares of common stock and (iii) performance stock ...

for children aged 1 to under 18 with pyruvate kinase (PK) deficiency who are not regularly transfused. The study met its primary endpoint of hemoglobin response, showing that 31.6% of children ...

Management is in the process of starting a pivotal mid-stage study on RP-L301 in patients with pyruvate kinase deficiency (PKD). With regard to CV indications, Rocket is also developing adeno ...

When discontinuing treatment, monitor patients for signs of acute hemolysis and anemia including ... a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first ...

In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia.

– ACTIVATE-Kids is the First Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency Who Are Not Regularly Transfused – – Safety Results Consistent with Safety ...

In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia.

AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced that the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 ...