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Future work should be focused and lean: enroll truly deficient, higher-risk patients, standardize formulations and dosing, and test event-driven hard endpoints aligned to mechanism, while embedding translational markers to confirm on-target biology

K., Khan, M

1 For example, rapid and dramatic correction of anemia with simple RBC transfusions has anecdotally led to increased intracranial pressure, seizure, and stroke in patients with SCD

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