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Drug Trial Offers Uncertain Start in Race to Save Children With Progeria

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Science  28 Sep 2012:
Vol. 337, Issue 6102, pp. 1594-1595
DOI: 10.1126/science.337.6102.1594

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This week marks the publication of a paper describing the first clinical trial of a drug for progeria, an ultrarare syndrome that seemingly ages children at warp speed. More than anything, the trial highlights just how difficult it can be to test a therapy in a terminal disease with so few patients. Twenty-five children, 75% of those known to have the disease worldwide when the trial began, received lonafarnib, a failed cancer drug that lab studies had indicated might help. The authors are upbeat about the trial's results, but they are in the minority.