Treatment of genetically defined rare diseases

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Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Second Quarter 2025
Globenewswire· 2025-07-29 18:48
― Announced results from the 12 mg dose cohort (n=16) of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD); pociredir was generally well-tolerated with no treatment-related serious adverse events (SAEs) ― ― Observed robust and rapid pan-cellular increases in fetal hemoglobin (HbF); meaningful improvements in key markers of hemolysis and anemia; encouraging trends in vaso-occlusive crises (VOCs) ― ― On track to provide clinical data from the 20 mg dose cohort by the end of 2025 ― ― Ended Q ...