Keith Fargo, PhD, on Potential Treatment Avenues for Charcot-Marie-Tooth


The chief scientific officer of the CMT Research Foundation discussed investigative cell and gene therapy approaches for treating CMT.

“There are resources available for Charcot-Marie-Tooth (CMT). So, if you as a physician have a patient you suspect may have CMT, there are genetic tests available. There are centers of excellence available. You can reach out to us at the CMT Research Foundation and we can help you with some of those resources. You can find us at, and especially for the physician scientists who are watching today, we have dollars to support your research.”

Charcot-Marie-Tooth disease (CMT) is a rare genetic disease comprising a collection of hereditary peripheral neuropathies. Those with the different types of CMT experience a gradual weakening of their sensory and motor muscles and no disease-modifying therapies exist today for CMT.

The CMT Research Foundation (CMTRF)’s mission is to support research that may lead to cures. The CMTRF funds a number of promising projects and research in CMT, many of which are taking cell or gene therapy approaches.

CGTLive spoke with Keith Fargo, PhD, chief scientific officer, CMTRF, to learn more about the CMTRF’s funded projects in the cell and gene therapy fields. He discussed promising work and partnerships, as well as resources available to those with CMT.

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