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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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Authors and Affiliations

  1. Philip J. Brooks and Steve Groft are at the Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.,

    Philip J Brooks & Steve Groft

  2. Danilo A. Tagle is at the Office of Special Initiatives, Office of the Director, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.,

    Danilo A Tagle

Authors
  1. Philip J Brooks
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  2. Danilo A Tagle
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  3. Steve Groft
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Correspondence to Philip J Brooks.

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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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