Organizations focused on Charcot-Marie-Tooth (CMT) disease have aligned with clinicians, and pharmaceutical companies to share a set of recommendations for how CMT clinical trials should be designed, with the recommendations published in Journal of the Peripheral...
The CMT Research Foundation is launching a new Journal Club webinar series designed specifically for researchers working in the CMT space. Each session will spotlight a recent, high-impact publication, with a short presentation from the research team followed by open...
The Peripheral Nerve Society hosted its annual conference in the Netherlands this June, and our research team went to learn about the latest advances, network with field leaders and accelerate progress for CMT treatments and cures. Here are our scientific team's...
https://youtu.be/AzOoaWPYpyc?si=44PoEHPxatjopWc- The first session of the CMT Research Foundation’s Gene Therapies Demystified series gave attendees the building blocks: what gene therapy is, why CMT is a compelling target, and what the science suggests about its...
One of the biggest obstacles in CMT research is not a shortage of potential therapies, but a delivery problem: nerves are extremely difficult to reach through the bloodstream. Dr. Kelly Langert's team at Loyola University Chicago took a creative approach, asking...
ENCell, a South Korean company focused on cell and gene therapies, announced in May 2026 (as reported by Korean outlet The Bio News) that the first patient has been dosed in a Phase 2a clinical trial of EN001, a treatment candidate for Charcot-Marie-Tooth disease Type...
https://www.youtube.com/watch?v=hrNkyIHed8U One of the most rapidly expanding frontiers in Charcot-Marie-Tooth research is also one of the most challenging to keep pace with. Now, the CMT Research Foundation is making it accessible to everyone. The CMTRF launched Gene...
The CMT Research Foundation has funded a new study at Johns Hopkins University to find a blood-based biomarker that could measure whether future CMT treatments are working. The research, targeted at subtype CMT2C, is led by Dr. Jeremy Sullivan and Dr. Charlotte...
ReviR Therapeutics, a previous CMT Research Foundation grantee, announced in March 2026 that the first human has been dosed with RTX-117, an experimental drug for Charcot-Marie-Tooth disease that ReviR says targets multiple CMT subtypes. The drug was administered in a...
Applied Therapeutics, who will be speaking at the 2025 Global CMT Research Convention, recently announced that they will be meeting with the U.S. Food and Drug Administration, during the third quarter of 2025, to discuss a potential New Drug Application for...