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Description

We initially anticipated that ABCB7 would employ a substrate-binding mechanism similar to that of ABCB6, in which a single porphyrin molecule is coordinated between two GSH molecules 40

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ACSL1, long-chain acyl-CoA synthetase 1

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mTORC1 hyperactivation arrests bone growth in lysosomal storage disorders by suppressing autophagy

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VWD illustrates how real-world data can help uncover diagnostic delays, quantify disease burden, and better understand patient journeys

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