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By week 16, a larger proportion of AXA1125-treated subjects (3540%) than those in the placebo group (825%) reached clinically relevant thresholds for MRI-PDFF reductions of 30%, ALT reductions of 17 IU/L, and cT1 reductions of 80 ms

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Fumarate hydratase inactivation in hereditary leiomyomatosis and renal cell cancer is synthetic lethal with ferroptosis induction

As a result, primary coenzyme Q 10 deficiency is a clinically heterogeneous disorder that includes five major phenotypes: (i) severe infantile multi-systemic disease, (ii) encephalomyopathy, (iii) cerebellar ataxia, (iv) isolated myopathy, and (v) nephrotic syndrome

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