You've probably heard of the drug Thalidomide, used to treat morning sickness in the 1950s which resulted in severe developmental defects (particularly phocomelia) in thousands of pregnancies. Despite causing one of the largest medical tragedies in recent history, Thalidomide still has medical applications today and only recently have researchers started understanding the mechanisms by which it causes birth defects.
Carl Zimmer has a nice article out in the NY Times (which I'd encourage you to read) on what we're learning about how Thalidomide causes birth defects, and the gains to be made from such insights.
[Hat tip to Carl for the heads up via his blog, The Loom]
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