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Rare-disease researchers pioneer a unique approach to clinical trials

Nature Medicine volume 29, pages 1884–1886 (2023)Cite this article

Randomization and placebo groups are giving way to biomarkers, real-world data from wearables, and multi-center and adaptive trials.

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    Mike May

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May, M. Rare-disease researchers pioneer a unique approach to clinical trials. Nat Med 29, 1884–1886 (2023). https://doi.org/10.1038/s41591-023-02333-4

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