Agios Pharmaceuticals said on Wednesday its sickle cell drug met one of its two main goals in a late-stage study of patients aged 16 and older, but failed to show a statistically significant reduction ...

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

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

While Mitapivat also showed a reduction in the primary endpoint of annualized rate of sickle cell pain crises (SCPCs) ...

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 ...

Shares in Agios Pharma lost nearly half their value in pre-market trading today after it posted mixed clinical results from a ...

India has launched BIRSA 101, the nation’s first indigenous CRISPR-based gene therapy for Sickle Cell Disease, marking a ...

Shares of Agios Pharmaceuticals sank after the company said a phase three trial of its mitapivat drug to treat Sickle Cell Disease missed some of its endpoints. The stock fell 34% to $30 after closing ...

Despite the mixed results, Agios intends to submit a marketing application for mitapivat in sickle cell disease after meeting with the FDA in the first quarter of 2026. The company also expects a ...

Science and Technology Minister Jitendra Singh on Wednesday launched India’s first indigenous CRISPR-based gene therapy for ...

A specialist from Oman, located at the crossroads of Africa and Asia, explains that his nation serves as a microcosm when it ...