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In the present study, we show the noninferiority of mitapivat versus splenectomy in 4.2 / mice with similar improvements in hematologic parameters and reduction in markers of hemolysis

Studies have shown that lyso-Gb3 accumulation impairs mitochondrial energy metabolism by interfering with the autophagy-lysosomal pathway, including the dysregulation of the mechanistic target of rapamycin (mTOR) signaling [50,51]

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