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It is not FDA-approved, is research-use-only, and is not for human consumption

In this section, we will discuss only quinones and nitrocompounds, compounds with oxidative mechanisms of action well-characterized

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These preclinical studies in a -thalassemic mouse model generated the rationale for a phase II proof-of-concept study (ClinicalTrials.gov NCT03692052) of mitapivat in patients with nontransfusion-dependent thalassemias (NTDTs), including both - and -thalassemia genotypes

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