Topline results from the RISE UP phase 3 trial demonstrated that mitapivat significantly improved hemoglobin response in patients with sickle cell disease.

AGIO faces uncertainty as the FDA misses its Dec. 7 deadline related to the sNDA for Pyrukynd in thalassemia and offers no new timeline.

Agios Pharmaceuticals has seen its fair value estimate cut from roughly $42.33 to about $32.13 per share, as analysts recalibrate expectations following mixed clinical outcomes and a more uncertain ...

Classical PKU (MIM # 261600) is the most frequent inborn error of metabolism and follows an autosomal recessive pattern of inheritance. Untreated patients ...

The firm will share updated data and new analyses from its Phase III trial with the agency, which issued a complete response ...

CAR-T cancer therapies now need to succeed in randomized, controlled trials to earn approvals. Elsewhere, a drug from Bristol ...

Maturity-onset diabetes of the young (MODY) is an autosomal dominant monogenic form of diabetes. This report describes a case with hepatocyte nuclear factor 1-alpha ( HNF1A )-MODY due to a novel ...