Findings showed 28.1% of patients treated with mitapivat achieved a TRR vs 11.8% of patients who received placebo. A phase 3 trial evaluating mitapivat in pediatric patients with pyruvate kinase ...
The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application for SL1009 (sodium dichloroacetate oral solution) for the treatment of pyruvate dehydrogenase complex ...
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