– Prespecified Statistical Criterion for the Primary Endpoint in ACTIVATE-KidsT Was Not Met; Results Were Clinically Meaningful, with Observed Response Rates Higher for Mitapivat than Placebo for the ...
– ACTIVATE-Kids is the First Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency Who Are Not Regularly Transfused – – Safety Results Consistent with Safety Profile for ...
Agios Pharmaceuticals' Rare Blood Disorder Drug Misses Primary Goal In Pediatric Study, Stock Slides
Mitapivat was generally safe and well-tolerated, with safety results consistent with the safety profile for mitapivat. Topline data from ACTIVATE-Kids study of mitapivat in children who are not ...
Findings showed 28.1% of patients treated with mitapivat achieved a TRR vs 11.8% of patients who received placebo. A phase 3 trial evaluating mitapivat in pediatric patients with pyruvate kinase ...
CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare ...
– Tebapivat Phase 1 Data in Sickle Cell Disease and Phase 2b Trial-in-progress Update in Lower-risk Myelodysplastic Syndromes will be Presented and Published – – Live and Webcast Investor Event with ...
CAMBRIDGE, Mass., Feb. 17, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, ...
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