Expanding rare disease drug trials based on shared molecular etiology

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Current clinical trial approaches in rare disease test one drug on one indication defined by a clinical phenotype. For targeted drugs, grouping patients by molecular etiology would make much more sense.

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Figure 1: Benefits of grouping by molecular etiology.

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Acknowledgements

P.J.B. is also a Program Director in the Division of Metabolism and Health Effects, National Institute on Alcohol Abuse and Alcoholism, National Institutes of Health, Bethesda, Maryland, USA.

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Correspondence to Philip J Brooks.

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The authors declare no competing financial interests.

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Brooks, P., Tagle, D. & Groft, S. Expanding rare disease drug trials based on shared molecular etiology. Nat Biotechnol 32, 515–518 (2014). https://doi.org/10.1038/nbt.2924

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