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Cheung WW, Zhou P, Zheng R, Gertler A, Oliveira EA, Mak RH (2024) Leptin signalling altered in infantile nephropathic cystinosis-related bone disorder

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As pharmacological agents may have more targeted mechanisms of action as compared to NPIs, precision approaches to combination interventions will need to be guided by an analysis of an individuals biological makeup, including, optimally, genetic risk factors and multiomics perturbations that have been linked to specific disease pathways or susceptibility