STAT+: Fulcrum study suggests improved efficacy for higher dose sickle cell pill
7.0
来源：
STAT
发布时间：
2025-12-07 14:00
摘要：
Fulcrum Therapeutics reported that a higher dose of its experimental pill, pociredir, significantly improved the efficacy in inducing fetal hemoglobin levels in patients with sickle cell disease. In an early-stage study, participants receiving 20mg daily showed an increase in fetal hemoglobin from 7.1% to 16.9%. This promising result boosts hopes for a simple and effective treatment for sickle cell disease, with findings presented at the American Society of Hematology annual meeting.
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关键证据
Higher dose of pociredir was more effective at inducing fetal hemoglobin.
Participants saw their levels of fetal hemoglobin rise from 7.1% to 16.9%.
Study presented at the American Society of Hematology annual meeting.
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AI评分总结
Fulcrum Therapeutics reported that a higher dose of its experimental pill, pociredir, significantly improved the efficacy in inducing fetal hemoglobin levels in patients with sickle cell disease. In an early-stage study, participants receiving 20mg daily showed an increase in fetal hemoglobin from 7.1% to 16.9%. This promising result boosts hopes for a simple and effective treatment for sickle cell disease, with findings presented at the American Society of Hematology annual meeting.