Expert Interview
Discussing Fulcrum's pociredir and other oral HbF inducers for sickle cell disease (SCD)
Ticker(s): FULCInstitution: Private Practice
- Professor Emerita of Medicine and Pediatrics, Albert Einstein College of medicine and former Director of the Sickle Cell Center for Adults at Montefiore Medical Center
- Treats 30 patients with sickle cell
- Research focuses on understanding mechanisms that lead to end organ damage in order to identify early biomarkers and targeted therapies
Of the different mechanisms being investigated, which of them seems to have the most promise moving forward?
Added By: max_adminCan you provide a broad overview of fetal hemoglobin (HbF) inducers and pyruvate kinase activators in development for sickle cell disease (SCD), including their mechanisms and available clinical data?
Added By: slingshot_insightsWhat are your thoughts on the efficacy of Fulcrum’s pociredir, particularly regarding its HbF increases at different dosage levels, and what level of increase would be needed to reduce fatigue and vaso-occlusive crises (VOCs)?
Added By: slingshot_insightsWhat are the safety concerns associated with HbF inducers like pociredir, particularly regarding potential cancer risks and other adverse events such as neutropenia and GI toxicity?
Added By: slingshot_insightsHow do you compare the potential of HbF inducers versus pyruvate kinase activators for treating SCD, and do you foresee one class being more successful than the other?
Added By: slingshot_insightsRegarding the kinetics of response, do you expect HbF induction to plateau within the 12-week treatment period, and could there be compensatory mechanisms that might reduce long-term efficacy?
Added By: slingshot_insightsAre You Interested In These Questions?
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