A research collaboration including scientists from the NIH’s (NCATS) and the University of Wisconsin–Madison, helped identify three promising molecular compounds from a collection of approved drugs to pursue as potential treatments for Charcot-Marie-Tooth disease (CMT), a genetic neurological disease for which there are no current treatments.
The nonprofit CMT Association initiated and supported the university research, and findings were reported on July 20, 2012, in the ACS Chemical Biology journal. The research team screened nearly 3,000 approved and investigational drugs from the NCATS Pharmaceutical Collection, in a laboratory test, or assay, for CMT. Identifying an already approved drug that possibly is effective for another disease can have many advantages over developing a medicine from the start, including shortening the time it may take to apply for human clinical trials.
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