Agios reported the results of the study on Wednesday, and they were mixed. Most patients didn’t experience much of a ...

Pyrukynd, which Agios sells for a rare type of anemia, missed one of its main objectives in a study seen as crucial for its ...

In its mission to grow the reach of its pyruvate kinase (PK) activator Pyrukynd (mitapivat), Agios Pharmaceuticals has come up short of producing an unequivocal win in the key indication of sickle | ...

Having endured an impoverished childhood and bare-bones sickle cell care, Edward ‘Donnell’ Ivy, MD, devotes his career to ...

PRNewswire/ - Medunik USA, a specialty pharmaceutical company, and part of Duchesnay Pharmaceutical Group (DPG), is dedicated to providing patients with rare diseases access to orphan drugs. Medunik ...

Trial met primary endpoint of hemoglobin response and key secondary endpoints of change from baseline in hemoglobin concentration and indirect ...

A community demands accountability after one Chicago mother and her young children were viciously jumped by middle schoolers— ...

Agios reported mixed results from a Phase 3 study of its drug Pyrukynd in sickle cell, raising doubts about the drug's ...

“I told God, ‘I don’t think it’s time yet. I don’t feel like I did enough for you,’” she recalled. A pastor’s wife later ...

If the gene therapy treatment is proven to be effective in curing the sickle cell disease, it will initially be made available to people with severe sickle cell disease, maybe with organ damage.

The Ghana National Association of Certificated Counsellors (GNACC) has officially signed a Memorandum of Understanding (MOU) with the Focus on Sickle Cell Foundation (FoSCel) to collaborate on ...