PerspectiveDrug Discovery

Power of Rare Diseases: Found in Translation

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Science Translational Medicine  04 Sep 2013:
Vol. 5, Issue 201, pp. 201ps11
DOI: 10.1126/scitranslmed.3006800

Figures

  • Fig. 1 Human biology drives drug discovery.

    (A) The strategy depicted is defined by a focus on diseases with the most severe unmet medical needs and in which mechanistic understanding of the biology is strong. (B) A PoC trial focuses on a population of patients in which the disease mechanism is as homogeneous as possible, often those with a rare genetic disease. When possible, we then extend findings to subsets of patients with more common disorders.

    CREDIT: V. ALTOUNIAN/SCIENCE TRANSLATIONAL MEDICINE