Working to Find a Cure for Charcot-Marie-Tooth: Keith Fargo, PhD
The chief scientific officer of the CMT Research Foundation discussed the foundation’s mission.
“People lose the ability to walk correctly, they begin to fall quite a bit. And then sometimes they lose the use of their hands as well. Braces can really help with keeping people able to do their activities of daily living and everyday tasks. But as of yet, there's really nothing that provides an effective treatment or a cure. And that's what the research foundation is here to provide.”
Charcot-Marie-Tooth disease (CMT) is a rare genetic disease for which no disease-modifying therapies exist today. CMT is a collection of hereditary peripheral neuropathies and those with the different types of CMT experience a gradual weakening of their sensory and motor muscles.
The CMT Research Foundation (CMTRF) was founded 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.
CGTLivespoke with Keith Fargo, PhD, chief scientific officer, CMTRF, to learn more about the CMTRF’s goals and mission. He discussed CMT and its disease burden on patients as well as the unmet needs in the disease.
Conservative Management Following Treatment With DMD Gene Therapy Elevidys
May 19th 2025Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia discussed a case he presented at the American Society of Gene and Cell Therapy’s 2025 meeting.
