Collaboration May Help Uncover Treatments for Rare Neurologic Disease

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.

English: Onion bulb formations in a nerve biop...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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About cmtny

Due to symptoms of Charcot-Marie-Tooth (CMT) neuromuscular disorder, I needed to leave my career as a pharmacist earlier than planned. With the support of the CMTA, I founded the Upstate NY CMT support and action group to help others with CMT. I started this blog to raise awareness of this genetic peripheral neuropathy, through sharing personal stories. In addition to writing and advocating for others with CMT, I also co-coordinate our Art de Cure gallery at CPO, where we have raised over $21,000 for CMT research so far.
This entry was posted in Charcot-Marie-Tooth disease, CMTA, Health CMT, human clinical trials, neuromuscular disease, peripheral neuropathy, stem cell research and tagged , , , , , , , . Bookmark the permalink.

2 Responses to Collaboration May Help Uncover Treatments for Rare Neurologic Disease

  1. Pingback: Guest Post for Medical Mystery: “Funny Name….Serious Disorder” | Living Well with CMT (Charcot-Marie-Tooth) Disorder

  2. Pingback: Hand tremor as predictor of calf cramp in CMT patients perplexes researchers | Living Well with CMT (Charcot-Marie-Tooth) Disorder

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