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ASH 2023 | Mitapivat in SCD: one-year follow-up results from the ESTIMATE trial

Myrthe van Dijk, MD, UMC Utrecht, Utrecht, The Netherlands, presents results from the Phase II ESTIMATE trial (EudraCT 2019-003438-18), investigating the safety and efficacy of one-year fixed-dose mitapivat treatment in patients with sickle cell disease (SCD). The trial found improved hemoglobin levels compared to baseline and decreased markers of hemolysis, as well as showing mitapivat treatment to be well tolerated with low grade treatment-emergent adverse events (AEs). This interview took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.

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Disclosures

Research Funding: Axcella Health Inc., Agios Pharmaceuticals Inc.