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Hope for Progeria - Successfully Validated on Mice, a Treatment Could Soon be Tested on Children
Date:6/30/2008

esis of this fatty acid. In fact, pharmacological molecules are known to block certain stages in this path of synthesis.

After several attempts they noted that a combination of a statin and an aminobisphosphonate could prevent the fixation of the fatty acid to the progerin, and thus reduce its toxicity. The progerin being less toxic, the disease develops more slowly.

Following these encouraging results, a clinical protocol based on this treatment piloted by Nicolas Levy in Marseille (France) is about to start up. It should last 3 years and will concern 15 of the 25 children affected by progeria in Europe. The aim of this protocol is to slow down the progression of the disease and, if possible, prolong the life expectancy - at present very limited - of the children affected.

Article published in Nature Medicine : http://dx.doi.org/10.1038/nm.1786


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SOURCE AFM-Association Francaise contre les Myopathies
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