Screen of the ReFRAME Compound Library for Therapeutic Agents to Prevent Red Blood Cell Sickling Using an Improved High Throughput Sickling Assay

Authors: Akito Nakagawa, Kaycie Morwood, Kristen Johnso, Lorenzo Berra, Lauren Boal, Sharl Azar, Mary Huang, Matthew M. Heeney, Donald B. Bloch, and Fumito Ichinose

Published: April 14, 2025

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An Ethical Allocation Scheme for Scarce Gene Therapies in Sickle Cell Disease andTransfusion-Dependent β-Thalassemia

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Clinical and Economic Burden of Managing Patientswith Sickle Cell Disease Receiving Frequent Red BloodCell Transfusions in the United States