Sclerostin: how human mutations have helped reveal a new target for the treatment of osteoporosis

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This article describes how the study of a rare monogenic high bone mass disorder called sclerosteosis has provided a new insight into the regulation of bone formation.

In the 1990s there was a tremendous mood of optimism among pharmaceutical scientists that identification of disease-associated variations in the human genome would result in a surge of new drug targets (the ‘gene-to-drug’ mantra). To date, the expected deluge of new drugs has not arrived. However, a small number of drugs arising directly from the study of rare human disorders showing Mendelian inheritance are now entering late stage clinical trials. Here we describe the advantages of this approach and discuss the background and early clinical trial findings with antibodies directed at a target identified in this way.


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