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

(Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering ... in children aged 1 to <18 years with PK deficiency who are not regularly transfused achieved ...

USA-based Agios Pharmaceuticals has announced that the ACTIVATE-Kids Phase III study of mitapivat in children aged 1 to <18 ...

Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) announced that the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to ...

Research and Development (R&D) Expenses: R&D expenses were $82.8 million for the fourth quarter of 2024, compared to $77.5 million for the fourth quarter of 2023, and $301.3 million for the full year ...

AGIO reports mixed fourth-quarter 2024 results. Its sole marketed drug, Pyrukynd, drives revenues year over year.

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT – Get Free Report) has been assigned a consensus recommendation of “Moderate Buy” from the twelve research firms that are currently covering the stock, ...

Despite promising Phase 3 results, safety concerns, namely liver injury, have impacted Agios stock and could affect Pyrukund's approval and market adoption. Read the full report here.

DCA will be used with a proprietary genetic test to treat an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD) 1. The FDA granted Priority Review ... DCA is a ...