Results from a phase 3 clinical indicate that the oral drug mitapivat is safe and effective for treating adults with pyruvate kinase deficiency, a genetic condition that causes red blood cell ...

The FDA has approved Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. The Food and Drug Administration (FDA) has approved Pyrukynd ® (mitapivat) ...

Please provide your email address to receive an email when new articles are posted on . The FDA approved mitapivat for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.

Rocket Pharmaceuticals Announces Updated Positive Preliminary Clinical Data from Phase 1 Trial of RP-L301 for the Treatment of Pyruvate Kinase Deficiency — Updated Data Demonstrate Sustained ...

Credit: Shutterstock. Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes. The Food and Drug Administration (FDA) has accepted for Priority ...

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood ...

Pyruvate kinase deficiency (PKD) is a genetic blood disorder that causes red blood cells to die prematurely. Regular blood transfusions have been the primary treatment for people with PKD, until now.

CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today ...

BOSTON – Researchers have published the results of a clinical trial that led the U.S. Food and Drug Administration to recently approve mitapivat for the treatment of adults with pyruvate kinase ...

—Rocket’s Largest Lentiviral Pipeline Program Addresses Unmet Need for an Estimated 3,000 to 8,000 Patients in the U.S. and Europe— NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: ...

—Preliminary Data from First Patient Demonstrates Initial Safety and Tolerability of RP-L301, Near Doubling of Hemoglobin to Normal Range and Additional Normalization of Hemolysis Markers— —Rocket’s ...