Tag Archives: Extreme Phenotypes

Leveraging Nature’s Experiments: Mitigating Drug Discovery Risks Through Human Genetics
May 8, 2014

Extreme clinical phenotypes caused by rare genetic variations have been the basis of some of the most exciting drug targets in today’s pharmaceutical pipeline: PCSK9 in cholesterol, sclerostin in bone diseases, Nav1.7 for pain.  The rare gene signatures underlying these

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