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Future efforts should initiate pilot experiments and observational studies to preliminarily evaluate efficacy, identify benefiting subpopulations, and define treatment endpoints, thereby providing foundations for subsequent large-scale RCTs
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However, take into account the relative impact of many biological parameters on the metabolic fate of drugs in the human body, sophisticated models are necessary
Successful clinical validation would not only provide a new treatment option but also retrospectively validate the CXXC5-targeting mechanism in humans, which would increase confidence in PTD-DBM as well