More than 3 million people around the world have Charcot-Marie-Tooth (CMT) disease, but even with its quirky name, CMT is still relatively unknown. A progressive, degenerative disease involving the peripheral nerves that branch out from the brain and spinal cord to...
By: Keith Fargo, PhD, Chief Science Officer The CMT Research Foundation is pleased to announce a new project with Samsara Therapeutics, a US/UK-based biotech company that is developing a novel class of drugs for CMT1A. CMT1A is caused by duplication of a stretch of...
By: Keith Fargo, PhD, Chief Scientific Officer We are happy to report that a CMTRF-funded project designed to test a PIKfyve inhibitor in CMT4B1, a particularly devastating form of the disease, has passed the first stage and is moving on to the next stage, a...
Published by CMT News (slightly edited for clarity) It was an unexpected $1 million gift, even for Kent and Michele Stahl. The Charcot-Marie-Tooth Research Foundation (CMTRF) was hosting a research convention in September when it announced Endgame, the...
Nearly every instance of CMT is caused by the mutation of a single gene and most genetic causes of CMT have been identified. The era of genetic medicine has already begun. The FDA first approved genetic medicines in 2016/2017. This set the foundation for a precision...
By Keith Fargo, Chief Scientific Officer At the CMT Research Foundation, we believe that more is achieved when people work together than when they work individually. This is especially true in biomedical research. Over the past few decades, a seismic shift has...
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