Earlier this year, the U.S. Food and Drug Administration (FDA) approved mitapivat (PYRUKYND) for the treatment of patients with hemolytic anemia due to pyruvate kinase deficiency (PKD). According to 2…
The FDA has recently approved a treatment called Pyrukynd as a therapeutic option for hemolytic anemia disorder sparked by pyruvate kinase deficiency. Hemolytic anemia is a condition where red blood…
Prior to February 2022, there were no approved disease-modifying therapies for those with pyruvate kinase (PK) deficiency. However, this has recently changed! According to a news release from biopharmaceutical company…
Rocket Pharmaceuticals, Inc. ("Rocket") has dedicated its mission to developing gene therapy solutions for rare childhood conditions. Recently, the company shared the publication of preliminary data from a Phase…
© Copyright Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
