Randomized Trialhigh evidence
Efficacy and safety of mitapivat in patients with sickle cell disease (RISE UP): a phase 3, double-blind, global, randomised, placebo-controlled trial.
Lancet (London, England)·October 24, 2026·PMID 42913682
Why it mattersSickle cell disease is driven by hemolytic anemia and vaso-occlusion, and current therapies have limitations. Mitapivat offers a novel oral mechanism targeting red cell metabolism, potentially improving anemia and reducing sickling. This trial provides high-level evidence on its efficacy and safety, informing treatment options for patients with recurrent pain crises.
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