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Sponsored Artificial Intelligence: A Modern Approach, Global Edition Artificial Intelligence: A Modern Approach, Global Edition

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Natalie Schmitz

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Review Open access Jul 2026

From Small Data to Big Decisions: How Clinical Pharmacology Shapes Rare Disease Development

Clinical pharmacology is expected to continue playing an important role in evaluating mechanistic plausibility, ensuring analytic rigor, and translating small datasets into meaningful evidence to inform development and regulatory decisions in rare diseases.

Yan Xu, Alissa Verone-Boyle, Natalie Schmitz et al. · 0 citations

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